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...
Videos
Rare disease spotlight
Read this inspiring article on how PhD student Katherine Gulliver’s personal experience of Williams Syndrome, influenced her further educational and professional paths. We are lucky to have Katherine as part of our Regional contacts team for Devon and Somerset. https://twitter.com/PlymUni/status/1265272895845986310
Our BA Education degree isn't just for future teachers.
Find out how the course inspired PhD student Kat Gulliver to study inclusive education abroad, and how her knowledge has supported her charity work with @HCPT and @WSF_UK
Find out more 👇
https://www.plymouth.ac.uk/courses/undergraduate/ba-education/katherine-gulliver
Have you signed up to be a part of #BeyondXLH, the Disease Monitoring Program for #XLH yet? http://www.BeyondXLH.com #StrongDataStrongerLives
This summer, we are pleased to introduce a series of leadership skill-building webinars for Young Adult Representatives of RDLA (YARR) members.
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