Exon Skipping

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
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 group of people from neuromuscular advocacy organizations gathered at the 2024 MDA Clinical & Scientific Conference.
Featured Article

Understanding Titinopathies

By Myrna Traylor | May 9, 2024
Illustration of a black metal pipeline on a yellow background
Featured Article

What Is the Drug Development Pipeline?

By Amy Bernstein | February 12, 2024
DNA spiral pill on black background. Genetically modified food concept.
Blog Post

DMD Gene Therapy Approval Ignites Hope

By Larry Luxner | August 1, 2023
Featured Article

How MDA Invests in Research Success

By Maggie Callahan | May 18, 2023
Closeup of an unclothed infant lying on its side on a white blanket while an adult hand holds a stethoscope to its chest.
Featured Article

Q&A: Understanding Congenital Muscular Dystrophy

By MDA Staff | February 16, 2023
Featured Article

Research Grants Create a Path to Progress

By Karen Doss Bowman | August 23, 2022
clinical-trial-alert-image
Blog Post

Clinical Trial Alert: Phase 2 Study of Oral Ifetroban in Individuals with DMD

By Sujatha Gurunathan | August 10, 2022
Scientific conference speakers
Featured Article

A Successful In-Person Conference

By MDA Staff | May 13, 2022