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News & Resources

Sarepta Therapeutics: SRP-9001 Gene Therapy Update

About SRP-9001 (AAVrh74.MHCK7.micro-dystrophin) SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Sarepta is responsible for global development and manufacturing of SRP-9001 and plans to commercialize SRP-9001 in the United States. In December 2019, the Company announced a licensing agreement […]

Catabasis: Discontinued Phase III PolarisDMD Trial

Catabasis has announced its Phase 3 PolarisDMD trial discontinuation with edasalonexent, an anti-inflammatory medication under investigation for Duchenne muscular dystrophy. The stop to the PolarisDMD trial was due to a lack of efficacy (no benefit compared to placebo) and not any safety issues. Catabasis is extremely disappointed by this outcome and thanks to the Duchenne […]

Solid Biosciences: Collaboration with Ultragenyx

Solid Biosciences announced a new strategic collaboration with Ultragenyx, a biopharmaceutical company with a successful track record of bringing novel therapies to patients with serious rare and ultra-rare genetic diseases. This new partnership is great news for our community as it will advance meaningful treatment options for Duchenne muscular dystrophy. Solid remains committed to its […]

Santhera Pharmaceuticals: Discontinued Phase III SIDEROS Study

Today Santhera Pharmaceuticals announced the discontinuation of their clinical program with idebenone, a molecule under investigation for people living with Duchenne experiencing respiratory decline and receiving corticosteroids. Read the full press release here. Data from their interim analysis concluded the study is unlikely to meet its primary endpoint. As a result, Santhera has decided to […]

Pfizer: FDA Fast-Track Gene Therapy Candidate

Last week, Pfizer announced that the FDA fast-tracked its investigational gene therapy candidate (PF-06939926) for Duchenne muscular dystrophy. This will help expedite the approval process in the US, and we hope it will also mean a faster submission to Health Canada. Read more about the announcement here. Pfizer is currently conducting Phase I and II […]

Jesse’s Journey: World Muscle Society (WMS) Conference 2020

The World Muscle Society (WMS) is hosting it’s 25th Annual Conference online. From September 28 – October 2, 2020, Our Director of Research and Advocacy, Nicola Worsfold, will be in attendance providing daily updates for our Duchenne community. DAY ONE: MONDAY, SEPTEMBER 28 Today included six industry-sponsored symposia, two focusing on Duchenne muscular dystrophy. Session […]

Catabasis Connection: October 2020

Catabasis has provided an update on edasalonexent, bone health, cardiac function, and more from their presentations at the Congress of the World Muscle Society. Click here to see the October 2020 – Catabasis Connection Edasalonexent: In Duchenne, the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and […]

Solid Biosciences: FDA Lifts Clinical Trial Hold

On October 1, 2020, Solid Biosciences announced that the FDA (U.S. Food and Drug Administration) has lifted the clinical hold placed on the company’s IGNITE DMD Phase I/II clinical trial, investigating SGT-001, a novel gene therapy for Duchenne muscular dystrophy. This trial was put on hold by the FDA last year due to a serious […]

Santhera Pharmaceuticals: Vamorlone Long-Term Data

On September 22, 2022, Santhera announced its publication of long-term data with vamorlone in Duchenne muscular dystrophy. This is data extended from their previously published 24-week data and continues to show a reduction in steroid associated safety issues such as stunting of growth, cushingoid appearance (rounded face), hirsutism (excessive hair growth), weight gain and behavioural […]

Catabasis Connection: September 2020

Catabasis has provided an update on how they’re preparing to share top-line results from the Phase 3 PolarisDMD trial. Click here to see the September 2020 – Catabasis Connection Edasalonexent: In Duchenne, the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. By […]

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