News

Today, Sarepta Therapeutics announced that the U.S. Food and Drug Administration (FDA) has approved AMONDYS 45 (casimersen). AMONDYS 45 is an antisense oligonucleotide from Sarepta’s phosphorodiamidate morpholino oligomer (PMO) platform, indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 45 skipping. AMONDYS 45 is Sarepta’s third […]

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Duchenne Data Foundation and the World Duchenne Organization start the Duchenne Emergency and Capacity Building Program Amsterdam, 15 February – In Rare Diseases like Duchenne and Becker Muscular Dystrophy (DMD/BMD), developing strong patient communities plays a fundamental role. Communities are able to act on a local level to have a global impact for the well-being […]

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Step by Step towards Duchenne FAIR data In 2019, we made a commitment towards making Duchenne data suitable for sharing by implementing the FAIR principles. In 2020, we commissioned FAIR Experts to support us in this endeavour. Today, we have come a long way but a lot more could be achieved with your active involvement. […]

The post Meeting on Data Sharing for Duchenne appeared first on World Duchenne.

Gene therapy uses viruses to deliver a micro-dystrophin gene or an exon skipping gene. COVID-19 is a virus. If I get vaccinated for COVID-19, will this have an impact on my possibility to have gene therapy treatment in the future? Short answer: no. The COVID-19 vaccine, just as any other mRNA vaccines will not impact […]

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All results of clinical trials generate new knowledge, and helps to advance the development of treatments. This includes the sharing of so-called negative results: study results that do not meet certain endpoints, or where efficacy is not seen. However, publishing negative data is important, as we can learn from this and not make the same mistakes […]

The post Lay summaries of SPITFIRE and THUNDERJET studies available appeared first on World Duchenne.

PTC Therapeutics today announced that Translarna™ (ataluren) has been granted marketing approval in the Russian Federation for nonsense mutation Duchenne muscular dystrophy (nmDMD). It is estimated that 13% of DMD patients have a nonsense mutation. In countries where it is approved, Translarna is currently the only medicine that targets the underlying cause of nmDMD. Read […]

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Today, Sarepta shared an update for its investigational PPMO SRP-5051 clinical trial study. MOMENTUM, the name of their phase 2 clinical trial peptide phosphorodiamidate morpholino oligomer study is for patients with Duchenne Muscular Dystrophy amenable to skipping exon 51.   Read the full press release here (external link) Highlights Results from the multiple-ascending dose trial demonstrate proof-of-concept for SRP-5051 and […]

The post Sarepta Announces Clinical Results from MOMENTUM study appeared first on World Duchenne.

Genethon is dedicated to design and develop a gene therapy products for rare diseases. On Monday 30th of November it received the authorisation from the ANSM, the French National Agency for Medicines and Health Products Safety, to start a clinical trial for the treatment of Duchenne muscular dystrophy with product GNT 004. This multicentre international […]

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Elizabeth Vroom shares her experience as mother of a Duchenne young man who recently celebrated his 30th birthday. Elizabeth Vroom is the mother of Justus, a 30-year-old adult living with Duchenne muscular dystrophy. Next to this she is also a prominent figure in the international Duchenne Community as Chair of the World Duchenne Organization. Duchenne […]

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On the second week of November, an international conference will take place virtually in order to present the patient registry of COVID-19 and rare diseases and to discuss diverse topics of interest related to the current pandemic. The International Congress on the Effects of COVID-19 on People with Rare Diseases will be held on November […]

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Every year our community heads to Washington, D.C. as part of PPMD’s Advocacy Conference (this year, being held virtually on March 10th — and there’s still time to register!) to communicate to Congress the need for continued...

The post Upcoming Webinars: Spotlighting Federally Funded Research Studies in Duchenne appeared first on Parent Project Muscular Dystrophy.

PPMD is excited to learn that the FDA has granted accelerated approval to AMONDYS 45™ (casimersen), a treatment of Duchenne in patients who have a confirmed mutation of the DMD gene that is amenable to...

The post FDA Grants Accelerated Approval to AMONDYS 45™ (Casimersen) appeared first on Parent Project Muscular Dystrophy.

When people think of dynamic duos and classic pairings, they probably think of Batman and Robin, Fred and Ginger, or Oreos and milk. For those of us in the Duchenne and rare disease community, we...

The post Senate Duchenne Champions Reintroduce BENEFIT Act Heading into PPMD’s Virtual Advocacy Conference appeared first on Parent Project Muscular Dystrophy.

Jared Sweet Golf Outing: The Perfect Socially-Distanced DIY Event! Sue and Jeff Sweet have held the Jared Sweet Golf Outing for 14 years. Thankfully they were able to alter their 2020 event and make it...

The post Golf Outings: The Perfect Socially-Distanced DIY Event! appeared first on Parent Project Muscular Dystrophy.

A variety of laboratory tests are often obtained in the assessment and treatment of Duchenne, and those laboratory tests can be incredibly confusing and variable in nature. As such, PPMD has developed a new resource...

The post New Resource! Common Labs and What They Mean in Duchenne appeared first on Parent Project Muscular Dystrophy.

Parent Project Muscular Dystrophy (PPMD) and Duchenne UK are excited to announce Professor Kanneboyina Nagaraju at Binghamton, the State University of New York, as the recipient of our Joint Research Grant Call of 2020. The...

The post Duchenne UK and PPMD Award $350,000 to Address Immunological Challenges of Gene Therapy in Duchenne appeared first on Parent Project Muscular Dystrophy.

Many years ago, PPMD developed a one-page resource of safe and unsafe medications in Duchenne. While many of those medications are now discussed in various related sections of our website, we recognize the need for...

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PPMD’s Annual Conference – Virtual Edition June 23-26, 2021 It’s hard to believe that we are still in this pandemic. We are getting closer to safer days ahead and we want our Duchenne and Becker...

The post Save the Date for PPMD’s Annual Conference: Virtual Edition – June 23-26, 2021 appeared first on Parent Project Muscular Dystrophy.

PPMD is thrilled to announce the members of the 2021 PPMD Adult Advisory Committee (PAAC)! PAAC members serve at a leadership capacity to represent the teen and adult Duchenne and Becker community. Despite the inability...

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Rally4RJ Virtual Walk in the Park: A DIY Virtual Walk Success Story! Suzanne Sullivan and her family were excited to hold their 2nd annual Rally4RJ 1 Mile Walk in the Park in the summer of...

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