Rare Diseases

Capricor Therapeutics Presents Phase 3 HOPE-3 Trial Results for Deramiocel in Duchenne Muscular Dystrophy at AAN 2026

Duchenne muscular dystrophy (DMD) · 3 min

Ractigen Therapeutics RAG-17 Shows 81% Reduction in ALS Biomarker in Phase I Trial

Neurology - Amyotrophic Lateral Sclerosis (ALS) · 3 min

Pharma Partnering US 2026: Biotech Dealmaking Outlook

biotech · 5 min

Kyverna's Miv-cel Shows Breakthrough Results in Stiff Person Syndrome Trial, Potentially First Approved Treatment

Neurology - Stiff Person Syndrome · 3 min

Ractigen Therapeutics RAG-17 Shows 81% Reduction in ALS Biomarker in Phase I Trial

Neurology - ALS (Amyotrophic Lateral Sclerosis) · 3 min

Small Molecule vs Biologics in Oncology: Market Trends & FDA Insights 2024

FDA · oncology · 9 min

Pasithea Therapeutics' PAS-004 Receives FDA Rare Pediatric Disease Designation for Neurofibromatosis Type 1 Treatment

FDA · 2 min

FDA Accelerated Approvals 2026: Zongertinib and Oncology Policy Shifts

FDA · oncology · 7 min

FDA Approval Adagrasib: Accelerated OK for KRAS G12C NSCLC Treatment

FDA · non-small cell lung cancer · 4 min

FDA Approves RGEN-101: RareGen’s Orphan Drug for Genetic Disorder

FDA · Rare Genetic Disorders · 7 min

Medera's AAV-SERCA2a Gene Therapy Receives FDA Fast Track Designation for Duchenne Muscular Dystrophy Cardiomyopathy

FDA · 2 min

Sedogen Receives U.S. Patent for Diazoxide Treatment of Prader-Willi Syndrome Behavioral Symptoms

2 min