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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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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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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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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, 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 (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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Italfarmaco S.p.A. has announced that the full results from the pivotal phase 3 EPIDYS clinical trial of givinostat in ambulant Duchenne patients six years of age and older have been published in The Lancet Neurology. Givinostat is Italfarmaco’s proprietary histone deacetylase (HDAC) inhibitor for the potential treatment of Duchenne. The EPIDYS clinical trial met its…

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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…

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I am traveling this week with my three sons who have Duchenne muscular dystrophy (DMD): Max, 18; Rowen, 15; and Charlie, 13. The four of us are in Denver for three days of clinical trial appointments and one day of cardiology appointments. Other Duchenne moms are often surprised that I can travel with my nonambulatory…

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NS Pharma recently joined PPMD for a community webinar to share an overview of two of the company’s new exon skipping trials: Brogidirsen (exon 44) and NS-050/NCNP-03 (exon 50). Vamshi Rao, MD, discussed these two new clinical trials for individuals with Duchenne who are amenable to either exon 44 or exon 50 skipping. The NS…

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