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FDA Accepts Translarna NDA Resubmission for Review
A Letter From PPMD’s Pat Furlong I am thrilled to share that the FDA has
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Flu Season Ready: Protecting Yourself and Your Family
With the start of school and the onset of winter, we can expect a rise
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FDA awards LAMA2-CMD therapy MDL-101 orphan drug status
MDL-101, Modalis Therapeutics’ epigenetic editing therapy for LAMA2-related congenital muscular dystrophy (LAMA2-CMD), has been granted
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Duchenne affects my entire life, down to a DIY project
I’m a mom to seven children: Lexi, 23; Max, 18; Chance, 17; Rowen, 15; Charlie,
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Mouse model reveals liver involvement in muscular dystrophy
A new mouse model mimicking the liver symptoms of myotonic dystrophy type 1 — the
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A new beginning of shared purpose at Shalom Medcare
Since September, my life has moved in an exciting new direction, starting with becoming a
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DM1 progression varies with sex, age at disease onset: Study
The progression of myotonic dystrophy type 1, known as DM1, differs according to sex and
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Dallas Community Comes Together to Raise $100,000 for CureDuchenne at Champions in Dallas Dallas’ Finest Unites to Advance Innovative Research and Care for Duchenne Muscular Dystrophy
Dallas, TX – October 22, 2024– The Dallas community came together in an extraordinary show
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Changing society and its views on disability, one column at a time
Like me, my colleagues here at Bionews, the parent company of this website, write about
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EMA agency continues to oppose renewing Translarna’s approval
The Committee for Medicinal Products for Human Use (CHMP) — part of the European Medicines
