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John Brandsema, MD, a pediatric neurologist at Children’s Hospital of Philadelphia, speaks with Healio about the importance of early diagnosis of muscular dystrophy.
Duchenne Muscular Dystrophy: diagnosis, clinical development and global researchMuscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, ...
Avidity's delpacibart etedesiran (del-desiran) was granted orphan drug status in Japan as a treatment for myotonic dystrophy ...
Emma Adair, 22, a student from Crumlin abseiled 120ft down the Europa Hotel in Belfast for charity. But her challenge doesn’t ...
Consultant Neurologist, Manipal Hospital, Kharadi, Pune breaks down the early warning signs of serious brain and nerve ...
Grey’s Anatomy” and “Euphoria” star Eric Dane has revealed his ALS diagnosis, a progressive neurodegenerative condition that ...
He notes people shouldn't be alarmed as many symptoms can overlap with other diseases. However, ALS Canada does have an online tool called ReferALS, with a checklist of early symptoms to encourage ...
With grace that extends far beyond the pageant stage, Miss Asia Pacific World 2013 Srishti Rana recently took to Instagram to champion a cause close to countless hearts—raising awareness for children ...
Aaron was born with muscular dystrophy and suffered a stroke in 2023, then spent much of the following year recovering in the Midwest because of Maui's limited medical resources. "It was beyond ...
Firefighters are in the street holding boots to collect donations. CTV’s Ainsley McPhail learns other ways you can donate to Muscular Dystrophy Canada ...
This muscle weakness has quite distinctive symptoms and causes. Recognize from the following explanation! Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle ...
More information: Dariusz C. Górecki et al, Is dystrophin immunogenicity a barrier to advancing gene therapy for Duchenne muscular dystrophy? Gene Therapy (2025). DOI: 10.1038/s41434-025-00531-y ...