treatment News

GAINESVILLE, Fla., and CAMBRIDGE, Mass., – Applied Genetics Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, which include X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM), announces two important initiatives to help patients with potential interest...
GAINESVILLE, Fla. and CAMBRIDGE, Mass. – Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare retinal diseases, today reported 12-month data from its ongoing achromatopsia (ACHM) Phase 1/2 clinical trials, including data from all adult...
GAINESVILLE, Fla. and CAMBRIDGE, Mass. – Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare retinal diseases, today reported additional positive data from the ongoing X-linked retinitis pigmentosa (XLRP) Phase 1/2 clinical trial, including 12-month...
LONDON — Privately owned UK biotech company Daval International Limited (http://www.davalinternational.com) has been informed by Australia’s Therapeutic Goods Administration (TGA) that its innovative anti-inflammatory agent AIMSPRO(R) has been awarded Orphan Status for the treatment of Amyotrophic Lateral Sclerosis (ALS). ALS is the most common form of Motor Neuron Disease (MND),...
NEWTON, Mass. – Alcresta Therapeutics, Inc., a leading commercial-stage company focused on developing and commercializing novel enzyme-based products, today announced  publication of a position paper by the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition (NASPGHAN) Pancreas Committee recommending the use of RELiZORB® (immobilized lipase cartridge), a first-of-its-kind digestive...
LEXINGTON, Mass. – Aldeyra Therapeutics, Inc. (Nasdaq: ALDX) (Aldeyra) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company for ADX-2191 (methotrexate for intravitreal injection) for the treatment of primary vitreoretinal lymphoma (PVRL). There are no approved treatments for PVRL, a rare, aggressive,...
BOSTON – Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the U.S. Food and Drug Administration (FDA) has approved the expanded use of ULTOMIRIS® (ravulizumab-cwvz) to include children (one month of age and older) and adolescents with paroxysmal nocturnal hemoglobinuria (PNH). ULTOMIRIS, a long-acting C5 inhibitor that offers immediate, complete and sustained...
BOSTON – Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced positive topline results from a Phase 3 study evaluating the safety and efficacy of ULTOMIRIS® (ravulizumab-cwvz) in adults with generalized myasthenia gravis (gMG). The study met, with high statistical significance, its primary endpoint of change from baseline in the Myasthenia Gravis-Activities of Daily Living...
INDIANAPOLIS — Eli Lilly and Company (NYSE: LLY) announced today that the European Medicines Agency’s (EMEA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for the use of ALIMTA(R) (pemetrexed for injection) as monotherapy for the maintenance treatment of locally advanced or metastatic non-small cell...