Clinical Trials

Inside a hospital room, two healthcare providers wearing scrubs, gowns, masks, and head covers stand beside a bed where a young boy is lying down.
Featured Article

Milestone Moment: A Look Back at the First Gene Therapy Trial for DMD

By MDA Staff | February 28, 2025
Medical illustration of musculature in the upper back, shoulders, neck, and head.
Featured Article

Stem Cell Innovations Drive Advances in Muscle Regeneration

By Andrew Zaleski | February 28, 2025
Composite image of scientific lab instruments: a microscope, a pipette, petri dishes, and a computer keyboard.
Featured Article

Innovations in GNE Myopathy Research

By Amy Bernstein | February 28, 2025
Blog Post

Clinical Trial Alert: Phase 2/3 Study of an Investigational Medication in Pediatric Patients with Generalized Myasthenia Gravis

By MDA Staff | February 19, 2025
Creatine kinase (CK), also known as creatine phosphokinase (CPK) or phosphocreatine kinase, is an enzyme expressed by various tissues and cell types. CK catalyses the conversion of creatine and uses adenosine triphosphate (ATP) to create phosphocreatine (PCr) and adenosine diphosphate (ADP). This CK enzyme reaction is reversible and thus ATP can be generated from PCr and ADP.
Blog Post

Simply Stated: RNA-Based Therapies in Development for Myotonic Dystrophy

By Sujatha Gurunathan | January 30, 2025
Titled book Amyotrophic Lateral Sclerosis along with medical equipment, Title of book invented and fictitious made in Photoshop, conceptual image
Blog Post

Simply Stated: Updates in Amyotrophic Lateral Sclerosis (ALS)

By Sujatha Gurunathan | October 29, 2024
Genetic engineeering concept with 3d rendering dna helix and a part of dna
Blog Post

Next-Generation Exon-Skipping Therapies May Change the DMD Landscape

By Myrna Traylor | September 13, 2024
A father, mother, and three sons sit on an outdoor patio step
Blog Post

My Gene Therapy Journey: The Curran Family

By Rebecca Hume | September 5, 2024
Parents hold their four-year-old daughter, who received Zolgensma for spinal muscular atrophy as an infant.
Blog Post

A Revolutionary Decade for Spinal Muscular Atrophy Therapies

By Chris Anselmo | August 1, 2024
Blog Post

Clinical Trial Alert: Phase 2 Pivotal Study of Sevasemten (EDG-5506) in Adults with Becker Muscular Dystrophy

By Sujatha Gurunathan | June 17, 2024