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FDA Grants Approval to DUVYZAT™ (Givinostat)
PPMD is excited to learn that the FDA has granted approval to DUVYZAT (givinostat), a histone deacetylase (HDAC) inhibitor indicated for individuals diagnosed with Duchenne muscular dystrophy from six years of age and older. DUVYZAT will be made available and marketed in the United States by ITF Therapeutics, LLC. DUVYZAT works by targeting pathogenic processes…
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Duchenne clinical trials show me how mature my sons have become
I recently took my three sons with Duchenne muscular dystrophy (DMD) to a hospital in Denver for three days of a clinical trial. One day was for each of them — Max, 18, Rowen, 15, and Charlie, 13 — and then they each had a day of cardiac appointments. The week was as successful as…
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FDA approves oral givinostat, as Duvyzat, to treat Duchenne MD
The U.S. Food and Drug Administration (FDA) has approved Italfarmaco’s givinostat, which will now be marketed as Duvyzat, as an oral suspension to treat people with Duchenne muscular dystrophy (DMD) ages 6 and older. Duvyzat becomes the first nonsteroidal therapy approved in the U.S. for patients with all genetic variants of DMD — the most…
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ITF Therapeutics Shares Update on DUVYZAT™ Following FDA Approval
Following the FDA’s approval of DUVYZAT (givinostat) on March 21, 2024, ITF Therapeutics, LLC, the company responsible for producing and marketing DUVYZAT in the United States, today shared an update on the drug and related resources. ITF Therapeutics has launched the DUVYZAT website, which provides Indication and Important Safety Information, a comprehensive list of FAQs,…
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These College Scholarships are Open to People with Disabilities
Going to college can be a life changing experience – from meeting new friends to expanding your knowledge to learning more about yourself. It can also be especially challenging while living with a rare condition like Duchenne muscular dystrophy. That’s where these scholarships aimed at individuals with Duchenne and those with other disabilities can come…
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PPMD’s 2024 Advocacy Conference Brings Together Community to Support Progress in Fighting for Every Future
Earlier this month, more than 120 PPMD advocates – people with Duchenne and Becker, parents, siblings, grandparents, and other family members and friends – from 26 different states gathered in Washington, D.C. to urge legislators in the U.S. Senate and House of Representatives to keep Duchenne and Becker at the forefront of their minds when…
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WATCH: Sarepta Therapeutics – Data update from MOMENTUM study, SRP-5051 (Webinar Recording)
Sarepta Therapeutics recently joined PPMD for a community webinar: Data Update From the MOMENTUM Study of SRP-5051. Sarepta Therapeutics was invited to share data from Part B of MOMENTUM, a Phase 2 Study of SRP-5051 in people living with Duchenne muscular dystrophy who are amenable to skipping exon 51. Sarepta shared efficacy findings, noting that…
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PTC Therapeutics Announces Plans to Resubmit Translarna™ NDA to FDA
PTC Therapeutics, Inc. has announced that based on recent feedback from the FDA, the company plans to re-submit a New Drug Application (NDA) for Translarna (ataluren), a dystrophin restoration therapy via stop-codon readthrough for the treatment of nonsense mutation Duchenne (nmDMD). PTC expects to make the NDA resubmission by mid-2024. PPMD previously held a webinar…
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Agamree, next-gen steroid, now available for DMD patients in US
Agamree (vamorolone) is now available for Duchenne muscular dystrophy (DMD) patients, ages 2 and older, in the U.S., where it will be dispensed through a specialty pharmacy network. The U.S. Food and Drug Administration (FDA) approved the next-generation steroid, expected to be more tolerable than standard DMD corticosteroids, in October. It will be marketed by…
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PTC Therapeutics Provides Key Regulatory Updates
PTC Therapeutics announced that, based on recent feedback from the FDA, by mid-year they plan to re-submit an NDA for Translarna (ataluren) for Duchenne caused by nonsense mutations (also called premature stop codons). We will keep the community updated on the progress. Read the full press release HERE The post PTC Therapeutics Provides Key Regulatory…
