Orphan drugs offer critical treatment options for rare diseases, improving quality of life for underserved patients and ...

By Daniella Parra March Biosciences was granted an orphan drug designation from the FDA to MB-105, its CD5-targeted CAR-T ...

MB-105, a first-in-class CD5-targeted CAR T-cell therapy, received FDA orphan drug designation for relapsed/refractory T-cell ...

据公开信息，该候选药物拟治疗的适应症是被称为全球五大绝症之首的运动神经元疾病——渐冻症，渐冻症又名肌萎缩侧索硬化症（ALS），著名社会活动“冰桶挑战”源于渐冻症绝症患者的家庭和支持者。 此前，士泽生物另一款人异体通用“现货型”中脑多巴胺能神经前体细胞注射液（“XS-411注射液”）IND 申请获得国家药监局受理。据了解，该候选药物的适应症是 帕金森病 。

While the former Biden administration showcased the Inflation Reduction Act as a key victory in the fight over high drug ...

Zydus has announced that the USFDA has granted Orphan Drug Designation (ODD) to Usnoflast, a novel oral NLRP3 inhibitor, for ...

Orphan Drugs Market Orphan Drugs Market Dublin, Jan. 22, 2025 (GLOBE NEWSWIRE) -- The "Orphan Drugs - Global Strategic ...

Biosciences receives US FDA orphan drug designation for MB-105 to treat relapsed/refractory CD5-positive T-cell lymphoma: Houston Thursday, January 30, 2025, 17:00 Hrs [IST] ...

KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has ...

再鼎医药宣布，美国食品药品监督管理局（FDA）已授予ZL-1310孤儿药资格认定；ZL-1310是一款高活性的具有同类首创潜力的DLL3 ADC，用于治疗小细胞肺癌。 孤儿药（Orphan ...

Zydus Lifesciences's Usnoflast receives Orphan Drug Designation from the USFDA for the treatment of Amyotrophic Lateral ...

Zydus Lifesciences announced that the USFDA granted Orphan Drug Designation to Usnoflast, a novel oral NLRP3 inhibitor, for ...