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Gene therapy SGT-003 wins FDA’s rare pediatric disease designation
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease status to SGT-003, […]
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Rollout expected by fall in US for new DMD therapy Duvyzat
ITF Therapeutics — which will be responsible for marketing Duvyzat (givinostat), Italfarmaco’s newly approved Duchenne muscular […]
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When life with Duchenne is hard, I remember the value of friendship
During the month of March, I took a break from writing my column because I […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
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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 […]
