The Manila Times3h
Nurix Announces U.S. FDA Orphan Drug Designation Granted to Bexobrutideg (NX-5948) for the Treatment of Waldenström Macroglobulinemia
Orphan Drug Designation follows positive Phase 1 data presented at the 12th International Workshop on Waldenström ...
inews.co.uk on MSN1d
The 3.5m people with rare diseases who face worsening treatment and diagnosis rates
Rare diseases affect one in 17 people in the UK, but a Government initiative designed to improve their quality of life will ...
American Council on Science and Health3d
“Rare” but Not Uncommon: The Hidden Crisis of Rare Diseases
Rare diseases - a medical condition so obscure that even your doctor has to Google. Despite their name, these diseases ...
Korro Bio gets Orphan Drug Designation from FDA for its genetic disorder treatment
Korro Bio received U.S. FDA's orphan drug designation for investigational medicine KRRO-110 to treat Alpha-1 Antitrypsin ...
Quoin Pharmaceuticals, Inc.: Quoin Pharmaceuticals Provides Corporate Update and Reports Fourth Quarter and Full-Year 2024 Financial Results
Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the "Company" or "Quoin"), a late clinical-stage specialty pharmaceutical company focused on rare and orphan ...
Healio4d
FDA grants fast track designation to X-linked retinoschisis treatment
The FDA granted fast track designation to ATSN-201 for the treatment of X-linked retinoschisis, according to a press release ...
Their flesh turns to bone, but a new treatment might help them
That is the predicament of patients with fibrodysplasia ossificans progressiva (FOP) or “Stone Man Syndrome”, a rare ...
Psychology Today6d
From Anecdotes to Data: Researching Sleep in a Rare Disorder
Anecdotal evidence has long suggested that sleep problems are especially common for people with Moebius syndrome. Now, a new ...
Opinion
BusinessWorld8dOpinion
Transforming rare disease outcomes
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: Upside In Rare Disease Innovation
PTC Therapeutics could boost revenue with vatiquinone for Friedreich's ataxia. Find out why PTCT stock is poised for growth ...
Pharmaceutical Technology11d
Beyond compliance: The strategic role of PASS in rare disease therapy success
Post-authorisation safety studies (PASS) are often seen simply as a regulatory necessity, a checkbox that ensures ongoing ...
Pharmaceutical Technology11d
Realising the opportunities for growth in the European orphan drug market
The European orphan drug market is growing and is already comparable in size and scope to the US market. The European Union ...