Rare diseases - a medical condition so obscure that even your doctor has to Google. Despite their name, these diseases ...
Investors are encouraged to read the Company's Annual Report on Form 10-K when it is filed with the Securities and Exchange ...
Anecdotal evidence has long suggested that sleep problems are especially common for people with Moebius syndrome. Now, a new ...
Orphan Drug Designation follows positive Phase 1 data presented at the 12th International Workshop on Waldenström ...
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 Ltd. (NASDAQ: QNRX) (the "Company" or "Quoin"), a late clinical-stage specialty pharmaceutical company focused on rare and orphan ...
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.
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.
That is the predicament of patients with fibrodysplasia ossificans progressiva (FOP) or “Stone Man Syndrome”, a rare ...
Korro Bio received U.S. FDA's orphan drug designation for investigational medicine KRRO-110 to treat Alpha-1 Antitrypsin ...
The FDA granted fast track designation to ATSN-201 for the treatment of X-linked retinoschisis, according to a press release ...
Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on ...