Rhythm Pharmaceuticals, Inc. has announced that it received orphan drug designation from Japan's Ministry of Health, Labour and Welfare for setmelanotide, a treatment aimed at acquired hypothalamic ...
SAN DIEGO, April 8, 2025 /PRNewswire/ -- Avidity Biosciences, Inc. (RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates ...
- Data from ongoing DELIVER trial demonstrated sustained functional improvement through 18 months; results from Registrational Expansion Cohort expected late 2025 - In Japan, Orphan Drug designation ...
Notice of Orphan Drug Designation for JR-446 for Mucopolysaccharidosis Type IIIB by Ministry of Health, Labour and Welfare of Japan MEDIPAL HOLDINGS CORPORATION(TSE 7459, MEDIPAL) and JCR ...
TOKYO, LONDON and BOSTON, Oct. 28, 2025 (GLOBE NEWSWIRE) -- Kyowa Kirin Co., Ltd., (TSE: 4151, President and COO: Abdul Mullick, "Kyowa Kirin") and Orchard Therapeutics announced that Japan's Ministry ...
KalVista Announces Orphan Drug Designation and NDA Submission for Sebetralstat in Japan for Hereditary Angioedema –Orphan drug designation paves the way for potential of sebetralstat to be first oral ...
- Data from ongoing DELIVER trial demonstrated sustained functional improvement through 18 months; results from Registrational Expansion Cohort expected late 2025 - WALTHAM, Mass., Sept. 29, 2025 ...
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