Orphan Drug Designation follows positive Phase 1 data presented at the 12th International Workshop on Waldenström ...
Rare diseases affect one in 17 people in the UK, but a Government initiative designed to improve their quality of life will ...
Rare diseases - a medical condition so obscure that even your doctor has to Google. Despite their name, these diseases ...
Korro Bio received U.S. FDA's orphan drug designation for investigational medicine KRRO-110 to treat Alpha-1 Antitrypsin ...
Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the "Company" or "Quoin"), a late clinical-stage specialty pharmaceutical company focused on rare and orphan ...
The FDA granted fast track designation to ATSN-201 for the treatment of X-linked retinoschisis, according to a press release ...
That is the predicament of patients with fibrodysplasia ossificans progressiva (FOP) or “Stone Man Syndrome”, a rare ...
Anecdotal evidence has long suggested that sleep problems are especially common for people with Moebius syndrome. Now, a new ...
Reivi Dela Cruz was only four years old when he was diagnosed with Neurofibromatosis Type 1 (NF1), a genetic condition that causes tumors (neurofibromas, which are usually non-cancerous) to grow on or ...
PTC Therapeutics could boost revenue with vatiquinone for Friedreich's ataxia. Find out why PTCT stock is poised for growth ...
Post-authorisation safety studies (PASS) are often seen simply as a regulatory necessity, a checkbox that ensures ongoing ...
The European orphan drug market is growing and is already comparable in size and scope to the US market. The European Union ...