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Congress Moves Forward on Newborn Screening
Last week, the US House Committee on Energy and Commerce’s Subcommittee on Health considered the Newborn Screening Saves Lives Reauthorization Act (HR 2507), a bill that would reauthorize the national…
The Ups and Downs of Accessible Air Travel
A little knowledge and preparation can get your travel plans off the ground
Tags: Accessible Air Travel, Thrive 365, Travel
These 7 Things Can Help You Understand Gene Targeting Therapy
In recent years, drug developers have been able to pair the ever-expanding pool of gene identifications with advances in gene-targeted therapies. Researchers look to treat genetic disease at its source.
The State of Gene Targeted Therapies
How these promising therapies are being studied and tested for neuromuscular diseases
Tags: Drug Development, Featured Content, Gene Therapy, MDA Clinical and Scientific Conference, Research
Pushing Limits
A hiker proves she is stronger than FSHD by taking on the toughest trek of her life
Tags: From Where I Sit, Staying Active
Ongoing Clinical Trials
Updates on some of the most promising studies
Tags: Clinical Trials, Drug Approval, Drug Development, Featured Content, Gene Therapy, MDA Clinical and Scientific Conference, Research
Guiding Treatment for Myotonic Dystrophy
A Q&A with neurologist Tetsuo Ashizawa, M.D.
Tags: Gene Therapy, Research, Spotlight
From Drug Discovery to Delivery
FDA Panel (left to right): Janet Woodcock, MD, FDA Center for Drug Evaluation and Research, and Peter Marks, MD, PhD, FDA Center for Biologics Evaluation and Research. FDA directors and…
The Case for Newborn Screening
Identifying neuromuscular diseases at birth can yield great benefits but faces great challenges
Care Across the Lifespan
Addressing sensitive topics contributes to higher quality of life