Phase II
ABVC Biopharma, based in California, has programs in Phase II trials to treat major depressive disorder, adult attention deficit hyperactivity disorder and myelodysplastic syndrome.
Fulcrum Therapeutics will launch a Phase III study of losmapimod in people with facioscapulohumeral muscular dystrophy later this year. Tonix wins Orphan Drug designation for Prader-Willi asset.
Shares of NuCana have plunged more than 57% in premarket trading after the company announced its Phase III biliary tract cancer study. See more facts here.
Healx brings the power of artifical intelligence (AI) to rare disease research with an ultimate focus on improving patient outcomes.
Recent gene therapy research has created a durable response in terms of producing normally-shaped red blood cells and reducing the pain of sickle cell disease.
The study, which has yet to be peer-reviewed, found the Pfizer-BioNTech vaccine was only about 12% effective for children ages 5 to 11 years against the Omicron variant.
The FDA rejected Gilead’s New Drug Application, citing Chemistry Manufacturing and Controls (CMC) problems associated with the compatibility of the drug with the proposed container vial.
The FDA declined approval for Amryt Pharma’s therapeutic oleogel-S10. The topical gel is intended to treat a group of rare skin diseases called epidermolysis bullosa.
The FDA lifts its clinical hold on Ocugen’s COVID-19 vaccine, and Ocugen announces a clinical trial for the treatment of inherited retinal diseases.
The company is conducting analyses to understand safety data that had been gathered prior to the pausing of the three studies, NCT04605159, NCT04980391 and NCT05229068.
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