Emma Ciafaloni, MD, FAAN, dives into the latest developments in gene therapy for muscular dystrophies, focusing on Duchenne muscular dystrophy, and discusses challenges, genetic causes, and the ...
Myotonic dystrophy type 1 (DM1) is the most common cause of adult-onset muscular dystrophy, a genetic disorder that leads to ...
Partnering with Parent Project Muscular Dystrophy to ensure timely access to the trial for eligible patients~15,000 children are living with DMD ...
Editor’s note: This is an automatically generated transcript. Please notify editor@healio.com if there are concerns regarding accuracy of the transcription. We still have maybe 15% or so of people who ...
Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.
Dyne Therapeutics started the new year with proof-of-concept clinical data for experimental genetic medicines addressing two rare muscle diseases—one of which has no FDA-approved treatments. Now the ...
Muscular dystrophy is a disease caused by mutations in the dystrophin gene. The muscles of muscular dystrophy patients weaken and atrophy over time. Heart and breathing muscles may also eventually be ...
A new gene therapy for Duchenne muscular dystrophy (DMD) has shown promise in not only slowing the progression of the disease but potentially even reversing the muscle damage, with human trials set to ...
A mother's persistence helps revive an all-but-abandoned drug class and could aid her son and thousands of others with ...
Becker muscular dystrophy (BMD) is a genetic disorder that causes progressive muscle degeneration and weakness. This can cause a person to have reduced mobility such as problems walking. BMD can make ...
Coordination of Care for Patients with Duchenne Muscular Dystrophy: Teamwork is Everything Duchenne Muscular Dystrophy: Speaking with Patients and Families About Advanced Care ...
Parents of children with the rare genetic disease Duchenne muscular dystrophy (DMD) are appealing to the government, the ...
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