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
We are so excited to feature an incredible lineup of community artists for PPMD’s Community Concert: Harmony Together tomorrow evening, November 22 at 7 PM EST! Meet our featured community artists: https://www.parentprojectmd.org/ppmds-community-concert-harmony-together/
High expression of CHML predicts poor prognosis of multiple myeloma http://dlvr.it/RmBsMv
Novartis is conducting a clinical trial in patients with sickle cell disease 16 years and older with chronic kidney disease. The Phase II STEADFAST clinical trial is now enrolling.