Gene Therapy

A family poses with Disney's Pluto
Blog Post

Insurance Denials for Gene Therapy Treatment Delay Access to Care

By Rebecca Hume | September 29, 2023
A young girl with blonde hair sitting on a doctor’s table as a doctor, a woman with brown hair, uses a reflex hammer on the girl’s knee. Both girl and doctor are smiling.
Featured Article

Advances in the Treatment of Friedreich Ataxia

By MDA Staff | August 8, 2023
Scientist examines DNA models in modern Genetic Research Laboratory.
Blog Post

MDA’s Legislative Efforts to Increase Access to Genetic Medicine

By Joel Cartner | August 7, 2023
DNA spiral pill on black background. Genetically modified food concept.
Blog Post

DMD Gene Therapy Approval Ignites Hope

By Larry Luxner | August 1, 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
Two men in dark suit jackets and ties stand in front of a large blue screen with words projected on it: MDA Clinical & Scientific Conference.
Featured Article

2023 MDA Clinical & Scientific Conference Makes an Impact

By MDA Staff | May 18, 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
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
Digital background depicting innovative technologies in genetic research
Blog Post

Simply Stated: Research Updates in Pompe Disease

By Sujatha Gurunathan | March 29, 2023