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An essay on choosing hope in life with a progressive, degenerative disease
I hate muscular dystrophy. In my darker moments, I want to scream into the void
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Young Leader Living with Muscular Dystrophy Champions Aiming High and Setting Goals
Harvard graduate Caroline LeMay’s education helped pave the way on her quest for success. Now,
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A Guide to RNA-Targeted Therapies
A Guide to RNA-Targeted Therapies The post A Guide to RNA-Targeted Therapies appeared first on
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RNA Therapies Offer Keys to Treating Genetic Neuromuscular Diseases
While DNA holds our genetic code, RNA plays a vital role in gene expression. Researchers
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Understanding Thymidine Kinase 2 Deficiency
Thymidine kinase 2 deficiency (TK2d) is a life-threatening form of mitochondrial myopathy. These diseases affect
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Expert Tips for Handling an Insurance Claim Denial for Gene Therapy
When Alison Joseph and William Small’s two youngest sons were diagnosed with Duchenne muscular dystrophy
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Considering a Clinical Trial? 4 Things to Know Before You Enroll
For people living with neuromuscular diseases, few things bring more hope than progress in research,
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What Is a VUS? Variants of Unknown Significance in Genetic Testing and Why They Matter
Chris Weihl, MD, PhD New genetic testing technologies are improving the diagnostic journey for many
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Roche stopping bone health program in Duchenne
Roche has decided to stop recruitment for their Phase 2 SHIELD DMD study of satralizumab in Duchenne,
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I have new criteria for when my sons participate in DMD clinical trials
As a caregiver to three sons living with Duchenne muscular dystrophy (DMD) and a mother
