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 ...
The Manila Times4d
Quoin Pharmaceuticals Provides Corporate Update and Reports Fourth Quarter and Full-Year 2024 Financial Results
Investors are encouraged to read the Company's Annual Report on Form 10-K when it is filed with the Securities and Exchange ...
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 ...
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 ...
PharmiWeb5h
Eneboparatide met primary endpoint of normalising…
High-level results from the CALYPSO Phase III trial showed that eneboparatide (AZP-3601), an investigational parathyroid hormone (PTH) receptor 1 agonist, met its primary endpoint with statistical ...
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 ...
pharmaphorum6d
Rare diseases and orphan drugs need digital innovation
No area could benefit more from this disruptive approach than the rare disease sector and the development of orphan drugs: an area of high unmet therapeutic need and large financial investment.
pharmaphorum3d
The biggest challenges facing rare disease pharma – and how the industry can tackle them
As Rare Disease day 2020 approaches, we take a look at the biggest challenges facing orphan drug developers and ask whether the future is bright or bleak for these difficult conditions.
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 ...
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 ...
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 ...
Korro Bio, Inc.: KRRO-110 Receives Orphan Drug Designation from U.S. FDA for Alpha-1 Antitrypsin Deficiency
Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on ...