Parent Project Muscular Dystrophy

A recent article in Slate addresses the difficulties of developing new therapies, and the importance of the words that we all use in talking about therapy development efforts.

2010-08-26T14:43:51Z

The Medical RevolutionWhere are the cures promised by stem cells, gene therapy, and the human genome?

By Emily YoffeUpdated Tuesday, Aug. 24, 2010, at 7:10 AM ET

Dr. J. William Langston has been researching Parkinson's disease for 25 years. At one time, it seemed likely he'd have to find another disease to study, because a cure for Parkinson's looked imminent. In the late 1980s, the field of regenerative medicine seemed poised to make it possible for doctors to put healthy tissue in a damaged brain, reversing the destruction caused by the disease.

NIH presents new funding opportunity for investigators. Check out these grants with fall application deadlines.

2010-08-25T15:23:03Z

NIH Funding Opportunities

Fall 2010

Below find information and links for three NIH funding opportunities for investigators.

Exceptional, Unconventional Research Enabling Knowledge Acceleration (EUREKA) (R01)

http://grants.nih.gov/grants/guide/rfa-files/RFA-GM-11-003.html

Solicits proposals for exceptionally innovative research on novel hypotheses or difficult problems, solutions to which would have an extremely high impact on biomedical or biobehavioral research. Support may be requested for up to $800,000 in direct costs (excluding consortium F&A) over a four-year period, not exceeding $250,000 (direct costs, excluding consortium F&A) in any one year.

Application Due Date: October 21, 2010

NIH Common Fund Transformative Research Projects Program (R01)

http://grants.nih.gov/grants/guide/rfa-files/RFA-RM-10-010.html

Solicits applications proposing groundbreaking, exceptionally innovative, high risk, original and/or unconventional research with the potential to create new scientific paradigms or challenge existing ones. Budget requests should be commensurate with project needs for up to a five-year project period. Up to one third of the budget for this FOA will be reserved projects exceeding $1 million dollars in direct costs.

Application Due Date(s): October 27, 2010

Scalable Assays for Unbiased Analysis of Neurobiological Function (R01)

http://grants.nih.gov/grants/guide/rfa-files/RFA-MH-11-040.html

Solicits applications to develop novel, robust analytical platforms using in vitro assays to reveal changes in neuronal and/or glial function. The goal is to adapt state-of-the-art measures of basic cellular processes or molecular events that are key mediators of brain function with the intent to probe mechanisms and/or perturbations in an unbiased and efficient manner. The novel assay platforms would provide opportunities to measure neurobiological endpoints and build a pipeline to be used in the context of target identification and drug discovery.

Application Due Date: November 17, 2010

BioMarin to Host Conference Call to Discuss Results of Phase 1 Clinical Study BMN 195 for Duchenne Muscular Dystrophy

2010-08-19T19:37:24Z

BioMarin to Host Conference Call to Discuss Results of Phase 1 Clinical Study BMN 195 for Duchenne Muscular Dystrophy

Patients, Families and Physicians Invited to Participate, Submit Questions

Earlier this month, BioMarin Pharmaceutical announced the completion of its Phase I clinical study of BMN-195, a small molecule utrophin up-regulator for the treatment of Duchenne muscular dystrophy (DMD), and the discontinuation of its BMN-195 drug development program. Although the company has terminated this program, it remains committed to this area of study. Company representatives will provide a brief summary of BMN-195 study results and answer questions on a conference call scheduled for Thursday, August 26, 2010. Interested parties are encouraged to submit questions via e-mail prior to the call: (sberg@bmrn.com).

Conference Call Details

Date: Thursday, August 26, 2010
Time: 10:00 a.m. Pacific | 6:00 p.m. GMT

Hosts:
Dr. Henry Fuchs, M.D.
Executive Vice President and Chief Medical Officer

Dr. Jackie Walling, M.D., Ph.D.
Vice President, Clinical Development

U.S. / Canada Dial-in Number: .............. 800-435-1398
International Dial-in Number:.................. 617-614-4078
Participant Code:.................................. 76407804

Contact:
Susan Berg
BioMarin Pharmaceutical Inc.
sberg@bmrn.com

www.bmrn.com

ACE-031 receives orphan designation in the US

2010-08-19T16:51:03Z

Acceleron Pharma Receives FDA Orphan Designation for ACE-031 for the Treatment of Duchenne Muscular Dystrophy

Download PDF
CAMBRIDGE, Mass.– August 19, 2010 - Acceleron Pharma, Inc., a biopharmaceutical company developing novel therapeutics that modulate the growth of cells and tissues including muscle, bone, fat, red blood cells and the vasculature, today announced the United States Food and Drug Administration (FDA) granted orphan designation for ACE-031 for the treatment of Duchenne Muscular Dystrophy (DMD), a fatal neuromuscular disease in which patients experience a progressive loss of muscle mass and strength. ACE-031 is an investigational protein therapeutic being developed to increase muscle mass and strength.

“In the past two weeks, the FDA has granted both orphan status and Fast Track designation to ACE-031 in recognition of the drug’s potential to address the enormous unmet medical need for therapeutics to treat DMD patients”, said Matthew Sherman, M.D., Chief Medical Officer at Acceleron. “We will continue to work collaboratively with clinical investigators, health authorities and patient advocacy groups around the world to develop ACE-031.”

Orphan drug designation is granted by the FDA Office of Orphan Products Development to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders. Under the Orphan Drug Act, the FDA may provide grant funding towards clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the United States following drug approval by the FDA. The approval of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval. For more information about orphan designation, please visit the FDA website at www.fda.gov

About ACE-031

ACE-031 is an investigational protein therapeutic that builds muscle and increases strength by inhibiting signaling through a cell surface receptor called activin receptor type IIB (ActRIIB). ACE-031 is a recombinant fusion protein that is produced by joining a portion of the human ActRIIB receptor to a portion of a human antibody. This creates a freely circulating, decoy version of ActRIIB which removes proteins, such as GDF-8 (myostatin), that limit the growth and regeneration of muscle. Animal studies with ACE-031 suggest that blocking signaling through ActRIIB leads to increased muscle mass and improved physical function in a range of animal models of muscle disease, including models of muscular dystrophy. ACE-031 is currently being studied in a phase 2 clinical trial of patients with Duchenne Muscular Dystrophy (DMD) who are also receiving corticosteroid therapy.

About Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy (DMD) is a debilitating and fatal genetic disorder characterized by the progressive loss of muscle strength and function. It primarily affects boys and occurs in approximately 1 in every 3,500 live male births. DMD is caused by genetic mutations that result in the absence or a defect in dystrophin, a protein necessary to maintain the structural integrity of muscle fibers. This condition leads to deterioration of and damage to skeletal muscles, which eventually become infiltrated by non-functional scar and fatty tissue. As a result, patients experience a relentless decline in muscle strength that impairs their ability to walk, breathe and live independently. Many patients spend the majority of their lives confined to wheelchairs and eventually lose all upper body function. Few patients survive beyond their late-20s when their heart and respiratory muscles weaken and eventually fail.

About Acceleron

Acceleron is a privately held biopharmaceutical company committed to discover, develop, manufacture and commercialize novel biotherapeutics that modulate the growth of red blood cells, bone, muscle, fat and the vasculature to treat musculoskeletal, metabolic and cancer-related diseases. Acceleron’s scientific approach takes advantage of its unique insight into the regenerative powers of the TGF-β superfamily of proteins. ACE-011 is in Phase 2 clinical trials in patients with anemia. ACE-031 is being studied in a Phase 2 clinical trial in patients with Duchenne Muscular Dystrophy and ACE-041 is being studied in a Phase 1 clinical trial in patients with advanced cancer. In addition, the company is developing several other new product candidates that increase muscle mass, control angiogenesis, inhibit fat accumulation and increase bone mass and strength. Acceleron utilizes proven biotherapeutic technologies and capitalizes on the company’s internal GMP manufacturing capability to rapidly and efficiently advance its therapeutic programs. The investors in Acceleron include Advanced Technology Ventures, Alkermes, Bessemer Ventures, Celgene, Flagship Ventures, MPM BioEquities, OrbiMed Advisors, Polaris Ventures, QVT Financial, Sutter Hill Ventures and Venrock. For more information, visit www.acceleronpharma.com

CONTACT:

Acceleron Pharma:
Steven Ertel, 617-649-9234
Vice President, Corporate Development

Paul Kidwell (Media)
Suda Communications LLC
617-296-3854

Action Alert!

2010-08-17T20:04:47Z

No one can do everything,

but everyone can do something.

Dear friends,

Today is a critical day for Duchenne. Actually every day is a critical day for Duchenne. And we know that often it may seem we are coming to you, the Duchenne community, to get involved and to take part in numerous things. That is because no one can do everything...We need your help in this fight to end Duchenne. We need you to join Coach To Cure MD.

By joining our team, by participating in even the smallest way, you help:

push critical research forward;
develop standards of care to ensure that your child is cared for properly; and
remind decision-makers in Washington that our boys count - that their lives matter.

Participating doesn't have to hard, it doesn't have to be time consuming. But we must work as a team if we are going to see success in our sons' lifetimes.

Today we are asking you to participate in Coach To Cure MD. This one-day national event has the power and the potential to put Duchenne on the map. What better venue than a sport where young men are at the peak of their athleticism, to show the disparity and educate the country about Duchenne muscular dystrophy. You don't have to like football. You don't have to understand it or even be a huge fan.

We just need the incredible commitment and passion that this community faces every challenge with. On September 25, over 400 hundred college teams, over 15,000 coaches, and over 3 million viewers will see the Coach To Cure MD patch, will hear the Coach To Cure MD fundraising ask, and will hear our families' stories...We need your help in being the ground force that drives this program.

To date Coach To Cure MD has raised over $500,000 for Duchenne. This year wouldn't it be great to raise that much in one day? It's not an unobtainable goal.

All it takes is you...

Any of these things - all of these things will help Coach To Cure MD grow. We wanted a day, we wanted a moment, we wanted the country to hear about Duchenne on a national scale. Here is our chance.

Join us and make Coach To Cure MD as powerful and as impactful as it can be.

Warmest thanks,

Kimberly Galberaith
Executive Vice President

One day. One team. One voice.

End Duchenne.

Santhera obtains European patent for use of Catena/Sovrima in the treatment of muscular dystrophies.

2010-08-17T20:04:13Z

LIESTAL, Switzerland - A pivotal Phase III study with Catena/Sovrima is currently ongoing to investigate the safety and efficacy of the drug in Duchenne Muscular Dystrophy. Santhera has partnered the commercial rights for the product in the EU and Switzerland to Takeda Pharmaceutical.

The patent granted covers the treatment or prevention of weakness and loss of skeletal muscle tissue as well as cardiomyopathy associated with certain forms of muscular dystrophies including Duchenne Muscular Dystrophy, one of the most common and devastating types of muscular degeneration. The patent protection in Europe lasts until 2026. In the United States, a similar patent is pending. In early 2007, both the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) granted orphan drug designation providing market exclusivity for 10 and 7 years, respectively, following marketing approval.

"The European patent for Catena®/Sovrima® in Duchenne Muscular Dystrophy is an important achievement and extends the protection considerably beyond the market exclusivity that goes together with the orphan drug status", commented Thomas Meier, Chief Scientific Officer of Santhera.

Santhera is currently conducting a pivotal Phase III study named DELOS (DuchEnne Muscular Dystrophy Long-term IdebenOne Study) in seven European centers and one in the United States. The primary endpoint of this 12-month, double-blind, placebo-controlled study is the change in respiratory function measured by peak expiratory flow. Additional respiratory parameters, muscle strength and motor function tests as well as quality of life assessments are secondary efficacy variables. Both the EMA and the FDA agreed that a single pivotal study could suffice for approval.

About Santhera
Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative pharmaceutical products for the treatment of severe neuromuscular diseases, an area of high unmet medical need which includes many orphan indications with no current therapy. Santhera's first product, Catena® to treat Friedreich's Ataxia, is marketed in Canada. Following positive clinical results in Leber's Hereditary Optic Neuropathy, the drug is currently prepared for regulatory filings for marketing approval. Catena® is also being investigated in a Phase III study in Duchenne Muscular Dystrophy. Commercial rights in Europe for Friedreich's Ataxia and Duchenne Muscular Dystrophy are licensed to Takeda Pharmaceutical. Santhera's second compound fipamezole has demonstrated efficacy in reducing levodopa-induced Dyskinesia in Parkinson's Disease. Phase III development and commercialization rights in the United States and Canada are partnered with Biovail. For further information, please visit the Company's web site at www.santhera.com

Catena® is a trademark of Santhera Pharmaceuticals. Sovrima® is a trademark of Takeda Pharmaceutical.

For further information, contact
Thomas Meier, Chief Scientific Officer
Phone: +41 (0)61 906 89 64
thomas.meier@santhera.com

Thomas Staffelbach, Head Public & Investor Relations
Phone: +41 (0)61 906 89 47
thomas.staffelbach@santhera.com

Disclaimer/Forward-looking statements
This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements.

Clinical development program update: A Phase II, double blind, exploratory, parallel-group, placebo controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability, and pharmacokinetics in ambulant subjects with Duchenne.

2010-08-13T16:25:25Z

Clinical development program update: A Phase II, double blind, exploratory, parallel-group, placebo controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability, and pharmacokinetics in ambulant subjects with Duchenne.

SENT ON BEHALF OF JOHN KRAUS, MD, PHD
and PADRAIG WRIGHT, MD, PHD
(GLAXOSMITHKLINE)

Details regarding several of the investigator sites for the following study have been posted to www.orpha.net (the portal for rare diseases and orphan drugs) today: Study DMD114117: A Phase II, Double Blind, Exploratory, Parallel-group, Placebo controlled Clinical Study to Assess Two Dosing Regimens of GSK2402968 for Efficacy, Safety, Tolerability and Pharmacokinetics in Ambulant Subjects With Duchenne Muscular Dystrophy Additional study details, including inclusion/exclusion criteria and study endpoints are posted on www.clinicaltrials.gov. Investigator site details for this study, taking place in Europe, Turkey and Australia, will also appear on www.clinicaltrials.gov in the near future.

Sincerely,

John E. Kraus, MD, PhD Padraig Wright, MD, PhD
john.e.kraus@gsk.com padraig.x.wright@gsk.com

PPMD awards $600K to Nationwide Children’s Hospital for gene therapy study. Dr. Jerry Mendell leading follistatin gene therapy.

2010-08-09T17:53:00Z

FOR IMMEDIATE RELEASE

CONTACT: Parent Project Muscular Dystrophy
Will Nolan: 201-944-9985

Parent Project Muscular Dystrophy Awards $600K
to Nationwide Children’s Hospital
for Gene Therapy Study

Dr. Jerry Mendell Leading Follistatin Gene Therapy

Middletown, OH – August 5, 2010 – Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), will award a $600,000 grant to Nationwide Children’s Hospital in Columbus, Ohio to conduct clinical testing of a promising gene therapy technique for muscle disease.

Investigators, led by Jerry Mendell, M.D., director of the Center for Gene Therapy in The Research Institute at Nationwide Children's Hospital with co-investigator Brian Kaspar, Ph.D, will inject a modified virus (vector) carrying the gene for the muscle growth-stimulating protein follistatin into the quadriceps muscles of volunteers with Becker muscular dystrophy and sporadic inclusion body myositis. The goal of the study is to verify that the procedure is safe and to document any increase in quadriceps muscle size and function. People with these diseases have overall muscle weakness but with particular weakness of the quadriceps muscle, which is important for standing and sitting. Preliminary studies in mice with muscular dystrophy and in non-human primates demonstrated that follistatin delivered in this manner can cause significant increases in the size of injected muscles. Improvements in the strength of the mice and non-human primates were documented.

PPMD funding for the project will cover the costs of manufacturing the clinical grade viral vectors, and the costs associated with the clinical testing. If the initial study is successful, the investigators will expand the research to a phase II study and will also make plans to test it in Duchenne muscular dystrophy and other muscle diseases. The first clinical studies are planned to start in early 2011.

“This is the first time a gene therapy approach has been used to supply genes that generically stimulate muscle growth rather than directly replacing missing muscle proteins, “ explains Sharon Hesterlee, Ph.D., PPMD Senior Director of Research and Advocacy, “Other applications could include the treatment of muscles that have been injured directly through accidents or indirectly through disuse. “

PPMD’s CEO and President, Patricia Furlong believes the work being done at Nationwide Children’s Hospital has huge potential. “Part of PPMD’s mission has always been to fund promising research in the fight to end Duchenne. Not only does the work Dr. Mendell is doing with follistatin show great promise, but if successful, it would help all patients living with Duchenne and Becker muscular dystrophy, as well as other muscular issues. It complements the other exciting therapeutic avenues that PPMD is funding as well.”

Dr. Mendell is extremely grateful to PPMD for the grant and acknowledges, “PPMD is dedicated to supporting research and researchers that are navigating new paths in the treatment of muscular dystrophy. Pat and the PPMD community have not just been supportive financially, but have been true partners in this project. It is refreshing to work with an organization that is willing to take risks on treatments like follistatin. We look forward to continuing this partnership and sharing our results with the community.”

This grant was made possible, in large part due to the generosity of Chris and Anna Stansbury and Robert H. Book,

About Duchenne and Becker

Duchenne, the most common form of childhood muscular dystrophy, is a progressive and fatal muscle disorder affecting boys and young men that causes the loss of muscle function, wheelchair dependency and a decline in respiratory and cardiac function. Becker muscular dystrophy, which is less severe than Duchenne, occurs when dystrophin is manufactured, but not in the normal form or amount.

About PPMD

Parent Project Muscular Dystrophy (PPMD) is a national not-for-profit organization founded in 1994 by parents of children with Duchenne and Becker muscular dystrophy. The organization’s mission is to improve the treatment, quality of life and long-term outlook for all individuals affected by Duchenne muscular dystrophy through research, advocacy, education and compassion. PPMD is headquartered in Middletown, Ohio with offices in Fort Lee, New Jersey. For more information, visit www.parentprojectmd.org.

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PPMD announces major grant awards and gears up for the best Coach To Cure MD event yet. Check out all the details in the August newsletter.

2010-08-05T20:36:35Z

PPMD Awards $600,000 Grant for
Gene Therapy Study
PPMD will award a $600,000 grant to Nationwide Children’s Hospital in Columbus, Ohio to conduct clinical testing of a promising gene therapy technique for muscle disease. Led by Jerry Mendell, M.D., investigators will inject a modified virus (vector) carrying the gene for the muscle growth-stimulating protein follistatin into the quadriceps muscles of volunteers with Becker and sporadic inclusion body myositis. PPMD funding for the project will cover the costs of manufacturing the clinical grade viral vectors, and the costs associated with the clinical testing. If successful, the investigators will expand the research to a phase II study and will also make plans to test it in Duchenne and other muscle diseases. The first clinical studies are planned to start in early 2011. Read the release.

Additional PPMD Grants
In addition to the grant awarded to Nationwide Children's Hospital, PPMD recently funded two additional projects. First, PPMD will provide a grant to clinical sites participating in Acceleron Pharma’s Phase 2 clinical study of ACE-031. These grants will help patients’ families cover travel costs to and from the study site, and hotel rooms and meals during study visits. Read more about these travel grants. In addition, PPMD recently announced it was giving a $20,000 grant to Brad Hodges, Ph.D. and his company, Prothelia, Inc. for his project titled, “Bridge Funds for Intravenous Protein Therapy for the Treatment of Duchenne Muscular Dystrophy." Click here to learn more about Dr. Hodges work.

Acceleron Receives FDA Fast Track Designation
Acceleron Pharma received FDA Fast Track designation for ACE-031 for the treatment of Duchenne. ACE-031 is an investigational protein therapeutic being developed to increase muscle mass and strength. Read the release. Additionally, PPMD President Pat Furlong has written a blog to help explain what the FDA term Fast Track means.

Acceleron Pharma receives FDA fast track designation for ACE-031 for treatment of Duchenne.

2010-08-04T15:38:06Z

Read the release

PTC Therapeutics provides an ataluren update for families of patients who participated in a clinical trial.

2010-07-30T16:36:11Z

PTC Provides Ataluren Update

PTC Therapeutics has provided an update on ataluren. In our last conversation with senior PTC officials, we were promised an update on next steps before the end of July. As you will read in the release, parents of patients who were treated with ataluren in a PTC clinical trial at a U.S. site should discuss the individual benefits and risks associated of continued use of low-dose ataluren with their clinical trial investigator.

READ THE RELEASE

Again, while this trial process has not been an easy process for affected families in the community to experience, we must commend PTC for fulfilling their promise to communicate with our community when new information arises.

We will continue to keep you posted, as more information becomes available.

7/30/10

PPMD announces grants for clinical study sites. The funds will help offset travel costs for participants in Acceleron Pharma’s Phase 2 clinical study of ACE-031.

2010-07-14T14:11:35Z

FOR IMMEDIATE RELEASE

CONTACT: Parent Project Muscular Dystrophy
Will Nolan: 201-944-9985

Parent Project Muscular Dystrophy Announces Grants For Clinical Study Sites

Funds Will Help Offset Travel Costs for Participants

Middletown, OH – July 14, 2010 – Patricia A. Furlong, Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), announced that PPMD will provide a grant to clinical sites participating in Acceleron Pharma’s Phase 2 clinical study of ACE-031. These grants will help patients’ families cover travel costs to and from the study site, and hotel rooms and meals during study visits. The first two grants have been awarded to Alberta Children’s Hospital and McMaster University.

Earlier this year, Cambridge, MA-based biotechnology company Acceleron launched a Phase 2 clinical study in Canada of ACE-031, a drug that promotes muscle growth. Previous studies in healthy volunteers have demonstrated that the drug increases lean mass and favorably affects markers of fat and bone mass.

The ACE-031 Phase 2 study is currently enrolling patients. Participants must be at least four years old and still walking. Additional details are available on Acceleron’s website: www.acceleronpharma.com and at Clinicaltrials.gov (refer to study identifier NCT01099761).

Ms. Furlong explained the reason for PPMD’s support of the ACE-031 trial with these grants. “When a family makes the decision to allow their child to participate in a clinical trial, they are making a huge decision. They are placing the health of their child in the hands of the researchers behind the project and they are allowing their child to take risks that will hopefully benefit the entire Duchenne community. We feel strongly in the work that Acceleron is doing and we wanted to help the study sites help the participating families, so that financial stresses are not added to the laundry list of things they have to worry about. The researchers behind these clinical trials are not the only pioneers in Duchenne – support must be given to the young people who participate.”

About PPMD

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Denver Mayor declares June 26, 2010 “End Duchenne Day” in honor of PPMD’s 16th Annual Connect Conference.

2010-06-18T14:01:53Z

FOR IMMEDIATE RELEASE

CONTACT:

Parent Project Muscular Dystrophy
Will Nolan: 201-944-9985

Denver Mayor Proclaims June 26 “End Duchenne Day”
Celebrating Parent Project Muscular Dystrophy’s
16^th Annual Connect Conference

Conference Includes Launch of First-ever Duchenne Therapeutic Development Meeting

Middletown, OH – June 18, 2009 – Patricia A. Furlong, Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy (Duchenne), announced that the Mayor of Denver, Colorado, John W. Hickenlooper, has proclaimed June 26, 2010 “End Duchenne Day” in recognition of PPMD’s 16^th Annual Connect Conference. Denver is playing host to the country’s largest Duchenne-specific, international conference, June 24 – 27, 2010.

The Proclamation from Mayor Hickenlooper reads: “The Connect Conference serves as a way to link the resources and needs of the Duchenne muscular dystrophy community to clinical care providers, policymakers, industry professionals, and the medical research fields.”

Check out PPMD’s June newsletter for the latest info on the upcoming Connect Conference, the Ataluren trials, and our summer advocacy initiative. And meet our Person of the Month, Robert Knight!

2010-06-04T17:26:39Z

Check out PPMD’s June newsletter for the latest info on the upcoming Connect Conference, the Ataluren trials, and our summer advocacy initiative. And meet our Person of the Month, Robert Knight!

PMD's Annual Connect Conference
Launches Scientific Track
This year's Connect Conference in Denver, June 24-27, 2010 will not only a boast a new host city, but an incredible new scientific conference: the Duchenne Therapeutic Development Meeting. Running parallel to our Connect Conference, this meeting boasts forty distinguished scientific speakers, representing eight biotech companies (including AVI, GSK, Acceleron, and PTC Therapeutics), and will be the most comprehensive Duchenne-specific, scientific meeting in the U.S. Attendees to the Connect Conference will have the opportunity to attend sessions at the scientific meeting, as well as interact with speakers. Learn more about this great new addition and check out the complete agenda for both Conference tracks.

Ataluren Update
Sharon Hesterlee, Ph.D., PPMD's Senior Director of Research and Advocacy, has put together a chart resulting from the survey parents were asked to fill out regarding the ataluren trials. Check out the data PPMD was able to compile based on your feedback. Plus, read Pat Furlong's blog about the recent update call with PTC Therapeutics.

FDA and NIH will co-sponsor a meeting titled Antisense Oligonucleotide (AON) Therapies in Neuromuscular Diseases, taking place on September 27 and 28, 2010 in Washington DC. PPMD will participate in this important meeting.

2010-06-04T17:25:29Z

FDA and NIH will co-sponsor a meeting titled Antisense Oligonucleotide (AON) Therapies in Neuromuscular Diseases, taking place on September 27 and 28, 2010 in Washington DC. PPMD will participate in this important meeting. Read more

AVI-4658 demonstrates first ever reported generation of greater than 50% dystrophin-positive muscle fibers in a patient following systemic administration in Duchenne.

2010-06-02T14:19:34Z

AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA-based drugs, today announced topline biopsy data from Study 28, the ongoing Phase 1b/2 clinical trial of AVI-4658, AVI’s lead drug candidate being developed as a systemically administered treatment for a substantial subgroup of patients with Duchenne muscular dystrophy (DMD), a genetic muscle wasting disease caused by failure to produce dystrophin. Topline biopsy data from the study demonstrated the first ever reported generation of new dystrophin-positive muscle fibers of more than 50% of normal in a patient with DMD following systemic administration of a drug. All patients in the two highest dose cohorts of the study demonstrated generation of new dystrophin-positive muscle fibers, although treatment responses varied across and within treatment groups. Generation of functional dystrophin is considered critical for successful treatment of DMD, and AVI intends further clinical evaluation of AVI-4658 to help optimize a dosing regimen to achieve more consistent improvements among patients. Continue reading the release.

Follow Up Open Conference Call re: ataluren on Thursday, May 27 at 5pm eastern

2010-05-24T15:56:01Z

PTC Therapeutics and PPMD have scheduled a call on May 27, 2010 at 5:00pm eastern. The call will include PTC President Stuart Peltz and Chief Medical Officer for the ataluren trial, Langdon Miller, MD.

Stuart and Langdon will update the community about ataluren and future plans. The conference call will be open to 150 callers. Lines will be muted and PPMD’s Sharon Hesterlee will conduct the interview and field your questions. The discussion will include analysis of data from the ataluren trial and a summary of regulatory challenges. Langdon will describe PTC’s plan for moving forward with ataluren and for patients to access ataluren either within a clinical trial protocol or through an expanded access program. PTC will also provide an estimate of the timeline for these next steps.

Click here to continue reading

The early bird special for PPMD’s Annual Connect Conference has been extended until May 24. Register today! And if you haven’t booked your hotel room, make sure you take advantage of PPMD’s room block at the Denver Marriott City Center. Rooms are filling up quickly don’t miss out on this great room rate!

2010-05-18T18:12:26Z

The early bird special for PPMD’s Annual Connect Conference has been extended until May 24. Register today! And if you haven’t booked your hotel room, make sure you take advantage of PPMD’s room block at the Denver Marriott City Center. Rooms are filling up quickly don’t miss out on this great room rate!

Check out this important article by PPMD President Pat Furlong and Barbara Godlew, highlighting how advocacy organizations function as a direct conduit to specialized patient populations and how these groups can provide a communication channel to reach thousands of potential clinical trial participants.

2010-05-18T16:50:27Z

Check out this important article by PPMD President Pat Furlong and Barbara Godlew, highlighting how advocacy organizations function as a direct conduit to specialized patient populations and how these groups can provide a communication channel to reach thousands of potential clinical trial participants.

PPMD will host a follow up call with PTC Therapeutics regarding the ataluren trials on May 27, 2010 at 5pm eastern

2010-05-11T20:47:24Z

Follow Up

Open Conference Call re: ataluren

To participate in this open conference call, please dial 866.740.1260 and enter 9449985. Anyone calling from outside of the U.S. or the Canada, click here for the appropriate dial-in number. There will only be space for the first 150 callers. The line will open at 4:50pm. We will not be able to record this phone call, but we will provide a detailed summary for those of you who cannot make the call.

To submit a question for this call, please email us before 5pm eastern on May 25 at info@parentprojectmd.org. We will compile your questions and address as many as we can in the time available. We realize that questions may arise during the call. However, because of the large number of participants we are anticipating, we will not be able to open the phone lines for questions. Therefore, during the call we suggest you be near a computer so you can email questions to info@parentprojectmd.org. We will address as many of those "live" questions as possible.

UPDATE: Please be aware that we will NOT be recording today's open conference call at noon eastern, as previously thought. We have just found out that several officials from PTC will be on today's call and available to answer the questions you have submitted. However, PTC's legal department will not allow the call to be recorded. PPMD will take thorough notes and will distribute these notes within the next few days.

Parent Project Muscular Dystrophy

A recent article in Slate addresses the difficulties of developing new therapies, and the importance of the words that we all use in talking about therapy development efforts.

The Medical RevolutionWhere are the cures promised by stem cells, gene therapy, and the human genome?

NIH presents new funding opportunity for investigators. Check out these grants with fall application deadlines.

NIH Funding Opportunities

Fall 2010

BioMarin to Host Conference Call to Discuss Results of Phase 1 Clinical Study BMN 195 for Duchenne Muscular Dystrophy

BioMarin to Host Conference Call to Discuss Results of Phase 1 Clinical Study BMN 195 for Duchenne Muscular Dystrophy

Conference Call Details

ACE-031 receives orphan designation in the US

Acceleron Pharma Receives FDA Orphan Designation for ACE-031 for the Treatment of Duchenne Muscular Dystrophy

Action Alert!

No one can do everything,

but everyone can do something.

One day. One team. One voice.

End Duchenne.

Santhera obtains European patent for use of Catena/Sovrima in the treatment of muscular dystrophies.

Clinical development program update: A Phase II, double blind, exploratory, parallel-group, placebo controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability, and pharmacokinetics in ambulant subjects with Duchenne.

SENT ON BEHALF OF JOHN KRAUS, MD, PHD and PADRAIG WRIGHT, MD, PHD (GLAXOSMITHKLINE)

PPMD awards $600K to Nationwide Children’s Hospital for gene therapy study. Dr. Jerry Mendell leading follistatin gene therapy.

Parent Project Muscular Dystrophy Awards $600K to Nationwide Children’s Hospital for Gene Therapy Study

PPMD announces major grant awards and gears up for the best Coach To Cure MD event yet. Check out all the details in the August newsletter.

Acceleron Pharma receives FDA fast track designation for ACE-031 for treatment of Duchenne.

PTC Therapeutics provides an ataluren update for families of patients who participated in a clinical trial.

PTC Provides Ataluren Update

PPMD announces grants for clinical study sites. The funds will help offset travel costs for participants in Acceleron Pharma’s Phase 2 clinical study of ACE-031.

Parent Project Muscular Dystrophy Announces Grants For Clinical Study Sites

Denver Mayor declares June 26, 2010 “End Duchenne Day” in honor of PPMD’s 16th Annual Connect Conference.

Denver Mayor Proclaims June 26 “End Duchenne Day”Celebrating Parent Project Muscular Dystrophy’s16th Annual Connect Conference

Check out PPMD’s June newsletter for the latest info on the upcoming Connect Conference, the Ataluren trials, and our summer advocacy initiative. And meet our Person of the Month, Robert Knight!

Check out PPMD’s June newsletter for the latest info on the upcoming Connect Conference, the Ataluren trials, and our summer advocacy initiative. And meet our Person of the Month, Robert Knight!

FDA and NIH will co-sponsor a meeting titled Antisense Oligonucleotide (AON) Therapies in Neuromuscular Diseases, taking place on September 27 and 28, 2010 in Washington DC. PPMD will participate in this important meeting.

AVI-4658 demonstrates first ever reported generation of greater than 50% dystrophin-positive muscle fibers in a patient following systemic administration in Duchenne.

Follow Up Open Conference Call re: ataluren on Thursday, May 27 at 5pm eastern

PPMD will host a follow up call with PTC Therapeutics regarding the ataluren trials on May 27, 2010 at 5pm eastern

Follow Up

Open Conference Call re: ataluren

SENT ON BEHALF OF JOHN KRAUS, MD, PHD
and PADRAIG WRIGHT, MD, PHD
(GLAXOSMITHKLINE)

Parent Project Muscular Dystrophy Awards $600K
to Nationwide Children’s Hospital
for Gene Therapy Study

Denver Mayor Proclaims June 26 “End Duchenne Day”
Celebrating Parent Project Muscular Dystrophy’s
16^th Annual Connect Conference