treatment of neonatal hypoxic-ischemic encephalopathy
All entries for: Rare Diseases
August 23, 2026
Pharmazz, Inc.
Orphan Drug Designation
Willowbrook
1-50 employees
Disease Area: Rare Diseases
August 22, 2026
Bryan Therapeutics
Orphan Drug Designation
Rockdale
1-50 employees
treatment of argininosuccinic aciduria
Disease Area: Rare Diseases
August 22, 2026
Eli Lilly and Company
Orphan Drug Designation
Indianapolis
1-50 employees
treatment of primary immune thrombocytopenia
Disease Area: Rare Diseases
August 21, 2026
Nationwide Childrens Hospital
Orphan Drug Designation
Columbus
1-50 employees
treatment of SLC6A1-related disease
Disease Area: Rare Diseases
August 20, 2026
Ouro Medicines Ltd.
Orphan Drug Designation
Altrincham, Cheshire
1-50 employees
treatment of pemphigus
Disease Area: Rare Diseases
August 19, 2026
Eloxx Pharmaceuticals
Orphan Drug Designation
Arlington, MA
1-50 employees
treatment of autosomal dominant polycystic kidney disease
Disease Area: Rare Diseases
August 18, 2026
Novartis Pharmaceuticals Corporation
Orphan Drug Designation
treatment of atypical hemolytic uremic syndrome
Disease Area: Rare Diseases
August 18, 2026
Mapp Biopharmaceutical, Inc.
Orphan Drug Designation
San Diego
1-50 employees
treatment of Nipah virus disease
Disease Area: Rare Diseases
August 17, 2026
Dynavax Technologies Corporation
Orphan Drug Designation
Emeryville
1-50 employees
Prevention of plague (Yersinia pestis)
Disease Area: Rare Diseases
August 17, 2026
ARCK Biologics, LLC
Orphan Drug Designation
Towson
1-50 employees
Treatment of systemic sclerosis
Disease Area: Rare Diseases