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Having the support of friends, old and new, makes all the difference
I received an overwhelming response to my last column, in which I grappled with feelings of uselessness when my facioscapulohumeral muscular dystrophy (FSHD) prevented me from helping in the way I would have liked to do. In this case, I was sitting at home while my wife, Wendy, was undergoing surgery. Many of my readers,…
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Trial will test gene therapy for limb-girdle muscular dystrophy type
Regulators in France and Italy have given Atamyo Therapeutics the green light to launch a clinical trial testing ATA-200, an investigational gene therapy for children with a type of limb-girdle muscular dystrophy called LGMD2C or LGMDR5. The multicenter, dose-escalation Phase 1b study (NCT05973630) — which will assess the safety, efficacy, and pharmacological and immunological properties…
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Managing motherhood and caregiving: Where is the beauty?
My mind is spinning like the wheels on my son’s power wheelchair in the freshly fallen snow. You did not read that wrong, and it’s not a hypothetical. Here in Nebraska, it snowed this last week of March. It feels like a second winter. As if the spring snow isn’t enough, life is going full…
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WATCH: Community Update with ITF Therapeutics – Introducing DUVYZAT™ (givinostat) (Webinar Recording)
ITF Therapeutics recently joined PPMD for a webinar to provide the Duchenne community with an opportunity to meet members of the ITF Therapeutics team to learn more about their organization and next steps to support the availability of DUVYZAT. ITF Therapeutics outlined next steps in the launch of DUVYZAT, including work on insurance policy education…
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Fiscal Year 2024 Appropriations Package Signed Into Law, Including Critical Funding for Duchenne and Becker
At the beginning of March, PPMD’s Advocacy Conference brought together more than 120 advocates from 26 different states in Washington, D.C. to advocate on behalf of the Duchenne and Becker community. This past weekend, the Fiscal Year 2024 appropriations package was signed into law, funding the U.S. federal government through September 31, 2024. Notable provisions…
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Mitochondrial dysfunction may be a treatment target in DM2
In myotonic dystrophy type 2 (DM2), muscle cells have dysfunctional mitochondria, the cell’s so-called powerhouse that’s key to energy generation. The results imply that treatments to boost mitochondrial function could be beneficial in DM2, the researchers said in “Multi-level profiling unravels mitochondrial dysfunction in myotonic dystrophy type 2,” which was published in Acta Neuropathologica. “While…
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Managing Femur Fractures in Duchenne Muscular Dystrophy
By Jennifer Wallace, PT and Doug Levine, PT Introduction Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that affects an estimated 1 in 5,000 live male births and is largely characterized by progressive muscle weakness. DMD itself, and even some of the current medical treatments, present challenges beyond muscle weakness. One that we have recently…
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Miami Community Comes Together for CureDuchenne, Raising Nearly $2.5M Since 2021
DJ Irie of the Miami Heat Headlines “Champions in Miami” to Help Find a Cure for Duchenne Muscular Dystrophy Miami, FL, March 25, 2024 – CureDuchenne and the Finazzo family of Miami hosted “Champions in Miami” on Saturday, March 9, 2024 at the Kimpton Surfcomber in Miami Beach, bringing together Miami’s top business leaders, entrepreneurs, influencers,…
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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 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…
