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European committee not in favor of renewing Translarna’s approval
A committee of the European Medicines Agency (EMA) has maintained its initial recommendation not to renew the conditional marketing authorization for Translarna (ataluren), PTC Therapeutics’ medication for Duchenne muscular dystrophy (DMD). The recommendation by the Committee for Medicinal Products for Human Use (CHMP) comes after the company appealed the negative opinion issued in September 2023…
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How I realized I was doing too much for my sons with DMD
I do too much. You might assume I’m talking about caregiving because you’re reading a column written by a caregiver. And in part, you’re right. But mostly, it’s an admission that I’ve been doing things for my three sons with Duchenne muscular dystrophy (DMD) that they can do themselves. My oldest son, Max, 18, is…
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Duchenne Newborn Screening Bill Passes Arizona Senate
Earlier this week, Duchenne parent and PPMD Advocate Jill Castle testified before the Arizona State Senate Health & Human Services Committee in support of Senate Bill 1020, which seeks to add Duchenne to Arizona’s newborn screening panel. PPMD, along with partner organizations, also signed onto a letter in support of SB 1020. We’re excited to…
