Latest News
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BOSTON – Rhythm (NASDAQ:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, announced recent progress in the clinical development of the company’s lead product candidate, setmelanotide, a first-in-class melanocortin-4 receptor (MC4R) agonist. “We are pleased to announce important progress across our clinical... -
Audentes Therapeutics (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases, today announced positive interim data from the first dose cohort of ASPIRO, a Phase 1/2 clinical trial of AT132 in patients with X-Linked Myotubular Myopathy (XLMTM). ASPIRO is a...
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Ridgeland, MS — Amicus Therapeutics (FOLD) has commenced the U.S. registration Phase 3 trial with its investigational drug, Amigal(TM) (migalastat hydrochloride) for the treatment of Fabry disease. The Company has reached agreement with the FDA on the key protocol design elements of the pivotal trial, including the use of the...
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Rare disease spotlight
Rare Disease advocates from (L) Kenya 🇰🇪, Tanzania 🇹🇿, Zimbabwe 🇿🇼, Ghana 🇬🇭
Creating awareness in Africa! https://twitter.com/cmutena/status/1291335970051305473
@georgemsengi @raddafoundation @Psalmy_Bizzle
This was a fun night!!!
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