rare

FDA Approves Apellis EMPAVELI (pegcetacoplan) as the First C3G and Primary IC-MPGN Treatment for Patients 12 and Older

Proven efficacy across all three key markers of disease—68% reduction in proteinuria, stabilization of kidney function, and substantial clearance of…

3 weeks ago

Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

July 18, 2025 16:30 ET  | Source: Ultragenyx Pharmaceutical Inc. NOVATO, Calif., July 18, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical…

4 weeks ago

Quetzal Therapeutics Debuts with Expert Leadership, High-Impact Pipeline and $50 Million of Committed Capital

CHICAGO, July 16, 2025 (GLOBE NEWSWIRE) -- Quetzal Therapeutics, a newly formed biopharmaceutical company, announced its official launch today with…

1 month ago

Rhythm Pharmaceuticals, Inc. Announces Proposed Public Offering of Common Stock

July 09, 2025 16:16 ET  | Source: Rhythm Pharmaceuticals, Inc. BOSTON, July 09, 2025 (GLOBE NEWSWIRE) -- Rhythm Pharmaceuticals, Inc.…

1 month ago

Morpheus Super Premium Whisky: Radico Khaitan Ltd. Expands the portfolio, initiates launch in Uttar Pradesh

NEW DELHI, July 1, 2025 /PRNewswire/ -- Radico Khaitan Ltd., one of the largest homegrown alco-bev companies announces the launch of…

1 month ago

Press Release: Rilzabrutinib granted orphan drug designation in the US for sickle cell disease

Rilzabrutinib granted orphan drug designation in the US for sickle cell disease Fourth orphan drug designation for rilzabrutinib in rare…

2 months ago

Press Release: Sanofi to acquire Blueprint Medicines, expanding portfolio in rare immunological disease and adding early-stage pipeline in immunology

  Sanofi to acquire Blueprint Medicines, expanding portfolio in rare immunological disease and adding early-stage pipeline in immunology Adds fast-growing…

2 months ago