Gene Therapy

3d illustration showing molecules passing an ion channel in cell membrane
Blog Post

Simply Stated: Updates in Ryanodine Receptor 1-Related Disorders (RYR-1-RD)

By Sujatha Gurunathan | May 29, 2025
Illustration of the neuromuscular junction, where nerves send signals to muscles.
Featured Article

Understanding Congenital Myasthenic Syndrome: Causes and Treatments

By Justine Savage | May 23, 2025
Illustration of a researcher in a white lab coat climbing a ladder shaped like a DNA helix.
Featured Article

Why MDA’s Kickstart Program Is a Game-changer for Gene Therapies

By Amy Bernstein | May 23, 2025
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
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
Medical Research Scientist Working on Desktop Computer with Gene Analysis Software in Modern Science Research Laboratory. Lab Engineers in White Coats Conduct Experiments in the Background.
Blog Post

Simply Stated: Emerging Therapies to Treat DMD

By Sujatha Gurunathan | December 20, 2024
A young boy in a batman t shirt holds up a sign that says HOPE in front of a red curtain
Blog Post

MDA CEO and President Dr. Wood Reflects on 2024: A Year of Milestones and Momentum

By Donald S. Wood, Ph.D., President and CEO, Muscular Dystrophy Association | December 5, 2024
An illustration of a doctor’s arm and hand holding tweezers and manipulating DNA.
Featured Article

Gene Editing Holds Promise as Viable Therapy

By Larry Luxner | November 18, 2024
2023 Clinical and Scientific Conference
Blog Post

MDA Kickstart: A New Hope for Ultra-Rare Neuromuscular Disease Treatments

By Sharon Hesterlee, Ph.D. | September 9, 2024
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In Case You Missed It…

By MDA Staff | September 8, 2024