PPMD's Blog – October 2016 Archive (11)

PPMD Designates Center for Duchenne Muscular Dystrophy at UCLA, a Certified Duchenne Care Center

PPMD Awards Twelfth Clinic Certification as Part of Effort to Ensure People with Duchenne Receive Optimal Care

 

The Center for Duchenne Muscular Dystrophy at the University of California, Los Angeles…

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Added by PPMD on October 26, 2016 at 3:02pm — No Comments

PPMD Designates Center for Duchenne Muscular Dystrophy at UCLA, a Certified Duchenne Care Center

PPMD Awards Twelfth Clinic Certification as Part of Effort to Ensure People with Duchenne Receive Optimal Care

 

The Center for Duchenne Muscular Dystrophy at the University of California, Los Angeles…

Continue

Added by PPMD on October 26, 2016 at 3:02pm — No Comments

Solid Biosciences Granted U.S. and E.U. Orphan Drug Designations for Lead Gene Therapy Candidate for Duchenne Muscular Dystrophy

Solid Biosciences and its subsidiary, Solid GT, announced today that the FDA and the European Commission have granted Orphan Drug designations for the company’s gene therapy candidate, SGT-001,…

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Added by PPMD on October 24, 2016 at 9:00am — No Comments

PTC Therapeutics Provides Regulatory Update on Translarna™

PPMD is disappointed to learn that the FDA denied PTC Therapeutics' first appeal of the refuse to file letter issued in February. As we understand it, this is a process and may involve additional appeals. PPMD will be…

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Added by PPMD on October 17, 2016 at 10:24am — No Comments

Connect with PPMD in 2017!

Can you believe 2017 is just around the corner? It will be here before you know it and PPMD is busy putting together an exciting list of events that will continue to connect and inform our Duchenne community. It’s already a packed new year!

 

Check out where your PPMD family plans to be in 2017 and mark your calendars!…

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Added by PPMD on October 14, 2016 at 9:30am — No Comments

PPMD Awards $239,000 Research Grant to Dr. Terence Partridge of Children’s National Medical Center

Parent Project Muscular Dystrophy (PPMD) has awarded a $239,000 grant to Dr. Terence Partridge, Professor of Systematic Integrative Biology, Research Center for Genetic Medicine at Children's National Medical Center. This grant will help Dr. Partridge and his team continue their work in understanding exon skipping technology for future trials in…

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Added by PPMD on October 13, 2016 at 11:31am — No Comments

Anthem and Exondys 51 (Eteplirsen)

PPMD is deeply troubled to learn that health insurer Anthem will not be covering Exondys 51, stating the following position:

“The use of eteplirsen is considered investigational and not medically necessary for all indications, including but not limited to the treatment of Duchenne muscular dystrophy.”

This is not an…

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Added by PPMD on October 7, 2016 at 2:30pm — No Comments

PTC Therapeutics Announces New Analyses that Support the Potential Benefit of Ataluren in Preserving Lung Function in Non-Ambulatory Nonsense Mutation Duchenne Muscular Dystrophy Patients

PTC Therapeutics, Inc. today announced new data supporting the potential benefit of ataluren in preserving lung function in non-ambulatory people with nonsense mutation Duchenne. PPMD continues to urge the FDA to provide PTC with a full review and advisory committee meeting.…

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Added by PPMD on October 6, 2016 at 9:15am — No Comments

Catabasis Completes Target Enrollment for Part B of the MoveDMD® Trial, a Phase 2 Trial of Edasalonexent (CAT-1004) for the Potential Treatment of Duchenne Muscular Dystrophy

Catabasis announced that target enrollment of 30 patients has been reached for Part B of the MoveDMD trial, a 12-week trial to assess the safety and efficacy of edasalonexent (CAT-1004) in Duchenne. Edasalonexent is an oral small molecule that the company believes has the potential to be a…

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Added by PPMD on October 4, 2016 at 11:00am — No Comments

New Research Validates Translarna's™ (ataluren) Mechanism of Action to Promote Readthrough of Nonsense Mutations and Produce Full-length Functional Protein

PTC Therapeutics announced that the Proceedings of the National Academy of Sciences (PNAS) has published new results further validating Translarna's™ (ataluren) mechanism of action to promote readthrough of premature stop codons resulting from nonsense mutations in…

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Added by PPMD on October 4, 2016 at 11:00am — No Comments

Sarepta Therapeutics and Summit Enter Into Exclusive License and Collaboration Agreement for European Rights to Summit’s Utrophin Modulator Pipeline for the Treatment of Duchenne Muscular Dystrophy

Today, Sarepta Therapeutics and Summit announced a license and collaboration agreement, which grants Sarepta the right to commercialize Summit's utrophin modulators in Europe should they receive approval. This includes ezutromid, which is currently in the Phase 2 PhaseOut DMD trial. PPMD will be hosting a community update call with…

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Added by PPMD on October 4, 2016 at 8:30am — No Comments

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