Drug Development

Telemedicine and human Liver recovery concept. Neutral color palette, copy space for text.
Blog Post

New Therapy Brings Hope for Late-Onset Pompe Disease

By Melissa Donahue | September 21, 2023
Blog Post

Deconstructing SMA Types and Treatments

By Sujatha Gurunathan | August 23, 2023
Blog Post

FDA Approves Sarepta’s ELEVIDYS for Treatment of DMD

By Sujatha Gurunathan | June 22, 2023
Featured Article

Gene Therapy Options Grow With New Drug Approvals

By Larry Luxner | May 18, 2023
Featured Article

How MDA Invests in Research Success

By Maggie Callahan | May 18, 2023
A female doctor or nurse concentrates while reviewing at a patient's test results.
Blog Post

The Value of Natural History Studies for Neuromuscular Disease

By Sujatha Gurunathan | May 4, 2023
Motor neuron connecting to muscle fiber, 3D illustration. A neuromuscular junction allows the motor neuron to transmit a signal to the muscle causing contraction. It is affected by toxins and diseases
Blog Post

Simply Stated: Research Updates in Emery-Dreifuss Muscular Dystrophy (EDMD)

By Sujatha Gurunathan | April 28, 2023
Green background with word Clinical Trial Alert and a picture of scientists in a lab
Blog Post

Clinical Trial Alert: Phase 3 Study of Pozelimab and Cemdisiran in Adults with gMG

By Sujatha Gurunathan | April 26, 2023
FDA approved Biogen’s Qalsody for treatment of SOD1-ALS.
Blog Post

FDA Approves Biogen’s Qalsody for Treatment of SOD1-ALS

By Sujatha Gurunathan | April 25, 2023
Banner announcing the Pompe Disease Symposium, with speaker Priya Kishnani, MD, MBBS, at the 2023 MDA Clinical & Scientific Conference in Dallas, Texas. Photo Credit Larry Luxner
Blog Post

At MDA Conference, Pompe Disease Researchers Stressed Importance of Newborn Screening

By Larry Luxner | April 14, 2023