Santhera Pharmaceuticals Releases Letter to DMD Community Announcing Temporary Suspension of Enrollment in BreatheDMD Expanded Access Program

On Nov. 19, Santhera Pharmaceuticals released a letter to the Duchenne muscular dystrophy (DMD) community announcing its decision to suspend enrollment of new participants in the BreatheDMD Expanded Access Program (EAP) until the SIDEROS clinical trial is fully enrolled. The full letter follows. SIDEROS clinical trial and BreatheDMD program update: Dear Duchenne community, In February 2018, Santhera . . .

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Solid Biosciences Releases Letter to DMD Community Announcing Hold on IGNITE DMD Trial Due to a Serious Adverse Event

Today, Solid Biosciences released a letter to the Duchenne muscular dystrophy (DMD) community regarding the decision by the US Food and Drug Administration (FDA) to place the company’s IGNITE DMD clinical trial on hold because the latest child dosed experienced a serious adverse event (SAE). The full letter follows. Letter to the Duchenne Community: Update . . .

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Roche Genentech Releases Letter to DMD Community Announcing Discontinuation of Clinical Trials Program for DMD

Today, Roche Genentech released a letter to the Duchenne muscular dystrophy (DMD) community regarding its decision to discontinue its DMD clinical development program. The full letter follows. Letter to the DMD community from Roche Genentech: Dear members of the Duchenne community, We are writing to share with you unfortunate news about our clinical development program . . .

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World Muscle Society Roundup: Clinical Trial Results of Treatments in Development for DMD, XLMTM, SMA, and Pompe Disease

Several biotech and pharma companies presented interim results at the 24th Annual Congress of the World Muscle Society (WMS) held earlier this month in Copenhagen, Denmark. This roundup covers a few of these updates. Catabasis Pharmaceuticals presented positive results from its phase 1/2 MoveDMD trial and open-label extension with edasalonexent in boys with Duchenne muscular . . .

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Capricor Therapeutics Announces Positive Interim Results from Phase 2 Study of CAP-1002 to Treat DMD

In July of this year, Capricor Therapeutics announced positive interim results from its phase 2 (HOPE-2) clinical trial evaluating CAP-1002, the company’s lead investigational therapy for Duchenne muscular dystrophy (DMD), to treat boys and young men in advanced stages of the disease. In that analysis, meaningful improvements in mid-level upper limb and hand function were . . .

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Five Questions with DMD Researcher Matthew Wood

Matthew Wood, PhD, professor of Neuroscience at the University of Oxford and director of both the Oxford Rare Disease Center and the MDUK Oxford Neuromuscular Center, was awarded an MDA research grant totaling $210,000 over two years to optimize the delivery of genetic therapies such as oligonucleotides to tissues for treating Duchenne muscular dystrophy (DMD). . . .

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ReveraGen Presents Positive Interim Results from Phase 2 Extension Study of Vamorolone to Treat DMD in Young Boys

At the World Muscle Society annual meeting held earlier this month in Copenhagen, Denmark, ReveraGen announced positive results from its ongoing phase 2 clinical trial of vamorolone for treating Duchenne muscular dystrophy (DMD) in boys. Building upon previously reported positive results, CEO Dr. Eric Hoffman presented data gathered after 18 months of treatment in the . . .

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2019 MDA Engage DMD Symposium Scheduled on Nov. 9

At MDA, we believe in the power of research and the importance of building relationships among families, clinicians, and the scientists making discoveries to advance treatments and therapies. We invite individuals living with Duchenne muscular dystrophy (DMD), as well as their caregivers and loved ones, to attend the MDA Engage DMD Symposium taking place Nov. . . .

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NS Pharma Submits New Drug Application to FDA for Viltolarsen to Treat DMD Amenable to Exon 53 Skipping

On Oct. 2, NS Pharma Inc. (a wholly-owned US subsidiary of Nippon Shinyaku Co. Ltd.) submitted a rolling New Drug Application (NDA) to the US Food and Drug Administration (FDA) for its exon-skipping therapy under development, viltolarsen (previously NS-065/NCNP-01), to treat Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. On Sept. 26, Nippon Shinyaku . . .

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In a Letter to the Duchenne Community, Santhera Announces Positive Results of Phase 2 Vamorolone Study, EMA’s Review of Puldysa to Treat Symptoms of DMD

This week, Santhera released a letter to the Duchenne muscular dystrophy (DMD) community regarding an update on Santhera’s DMD drug development programs and its partnership with ReveraGen. The full letter follows. Letter to the DMD community from Santhera: Dear Duchenne community, It was great to connect with so many of you at the Parent Project . . .

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