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
So, the Burrells have totalled 20.3 miles today... 10 miles on Ian's bike has resulted in jelly legs for Sue, a 7.2 mile walk by the 3 of us and Ian did 3.1 miles at football meaning we've only got 37 miles and 2 days to go!!!
Please sponsor us if you can! https://twitter.com/BPA_Porphyria/status/1310255589948260356
The BPA'S, Sue Burrell, husband Ian & 3 Yr old daughter Abi, vowed to walk/run/cycle 321 miles to raise funds for the BPA in Aug/Sept. They've got just 57.3 miles to go in 3 days!! Please help support them, if you can, by donating here: https://www.justgiving.com/fundraising/Burrell321
Thank you so much
This is something everyone should read and retweet far and wide for the benefit of #RareDiseases and all other. It is the #truth about #COVID19. CheckOrphan fully supports this #letter and the signers efforts @RareDiseases @EveryLifeOrg @GlobalGenes https://www.aier.org/article/open-letter-from-medical-doctors-and-health-professionals-to-all-belgian-authorities-and-all-belgian-media/amp/?__twitter_impression=true
Incredibly hopeful news from @NataliesWish 💫 https://twitter.com/NataliesWish/status/1310592359130902529
Jordan Janz - the first patient in Dr. Cherqui's stem cell clinical trial - has not taken cysteamine for an entire year. This means that the transplanted cells are doing their job! Thank you, Jordan and all of our patient volunteers, for giving us hope for a brighter future.
