Rare Diseases

Rhythm Pharmaceuticals' IMCIVREE Receives European Commission Approval for Acquired Hypothalamic Obesity

Rare neuroendocrine diseases / Obesity · 3 min

Atara Biotherapeutics Receives FDA Complete Response Letter for EBVALLO Cancer Treatment, Faces Securities Class Action

Oncology - Post-transplant lymphoproliferative disease · 3 min

Cadrenal Therapeutics CAD-1005 Gets FDA Guidance for Phase 3 Trial in Heparin-Induced Thrombocytopenia

FDA · Hematology/Thrombotic Disorders · 3 min

Teva Acquires Emalex Biosciences for $700M to Gain First-in-Class Tourette Syndrome Drug Ecopipam

Pediatric Neurology - Tourette Syndrome · 3 min

Purespring Therapeutics Publishes Study on NPHS2 Genetic Variants Driving Adult-Onset FSGS in US and UK Populations

2 min

Neurocrine Biosciences INGREZZA Shows Clinically Meaningful Improvement in Tardive Dyskinesia Study

2 min

Novo Nordisk's Denecimig Shows Significant Bleeding Reduction in Hemophilia A Phase 3 Trial Published in NEJM

Hemophilia A · 3 min

IgA Nephropathy Market to Reach New Heights by 2036 as Novartis Zigakibart and Novel Immunotherapies Drive Growth

2 min

FDA Accelerated Approvals Oncology: 2026 Policy Review & Zongertinib Case

FDA · oncology · 7 min

Teva Acquires Emalex Biosciences for First-in-Class Tourette Syndrome Drug Ecopipam

2 min

Rein Therapeutics LTI-03 Phase 2 Trial Enrolls 8 Patients for Idiopathic Pulmonary Fibrosis Treatment

3 min

Expression Therapeutics Receives FDA Fast Track and Rare Pediatric Disease Designations for Hemophilia A Stem Cell Therapy

FDA · 2 min