Hospital for Special Surgery

When to See a Pediatric Orthopedist

Knowing when to book an appointment with a pediatric orthopedist instead of a pediatrician can help a child get faster ...
Healthline

What to Know About More Common Pediatric Musculoskeletal Disorders

Musculoskeletal disorders affect the bones, joints, muscles, and connective tissue. Many musculoskeletal disorders may occur in children, and some types are more common than others. Musculoskeletal ...
Nasdaq

Edgewise Receives Orphan Drug and Rare Pediatric Disease Designations for Its Muscular Dystrophy Program from FDA

BOULDER, Colo.--(BUSINESS WIRE)-- Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the U.S. Food & Drug Administration (FDA) has ...
Nasdaq

Solid Biosciences Receives Rare Pediatric Disease Designation from the FDA for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003

CHARLESTOWN, Mass., April 01, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, ...
Seeking Alpha

Atossa Therapeutics Receives FDA Rare Pediatric Disease Designation for (Z)-Endoxifen for Duchenne Muscular Dystrophy

Designation expands (Z)-Endoxifen program into rare pediatric neuromuscular disease and may qualify Atossa for a future Priority Review Voucher upon approval SEATTLE, Dec. 11, 2025 /PRNewswire/ -- ...
Business Insider

Wave Life Sciences Receives FDA Rare Pediatric Disease Designation for WVE-N531 for the Treatment of Duchenne Muscular Dystrophy

Designation highlights significant unmet needs in DMD; dystrophin data from potentially registrational FORWARD-53 trial of WVE-N531 are on track for 3Q 2024 In previous Part A trial, WVE-N531 achieved ...