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Building routines that support Duchenne care
Shalom Lim, who lives in Eastern Singapore, was diagnosed with Duchenne muscular dystrophy at 4
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Setting expectations for treatment adherence in Duchenne care
Edward Smith, MD, a neurologist, explains why setting clear expectations around treatment benefits and risks
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Simply Stated: Introduction to CIAO1-Related Neuromuscular Disorder
CIAO1-related neuromuscular disorder is an inherited condition that was first reported on in late 2023.
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EU panel recommends expanded use of DMD treatment Agamree
The Committee for Medicinal Products for Human Use (CHMP) has recommended that approval of Agamree
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Advancing Duchenne Newborn Screening: A Collaborative Path Forward
On April 24, 2026, PPMD, in collaboration with the Muscular Dystrophy Association (MDA) and the
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Expert Advice for Preparing for Medical Emergencies with NMD
Julianne Meiser, MSS, LCSW, an Outpatient Social Worker in the Division of Neurology at the
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Starting corticosteroids earlier helps improve motor function in DMD
Starting daily corticosteroids earlier in young children with Duchenne muscular dystrophy (DMD) helps them move
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A tribute to a friend who taught us how to love, lead, and show up for others
Last Saturday, I lost a dear friend of mine, Timothy Chan, who had Duchenne muscular
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Patient-led group launches network of clinics to improve LGMD care
Patient-led nonprofit The Speak Foundation has announced the launch of the LGMD Centers of Excellence,
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Clinical Research Alert: Phase 3 Study of Omaveloxolone in Children and Teenagers with Friedreich Ataxia (FA)
Researchers at Biogen are seeking pediatric FA patients to participate in a phase 3 clinical
