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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
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Roche Announces Termination of Phase 2 SHIELD DMD Study
We are disappointed to learn the news that Roche has made the difficult decision to
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New gene therapy for OPMD shows lasting success in small US trial
Four people with oculopharyngeal muscular dystrophy (OPMD), a disease type marked by swallowing difficulties, experienced
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MDA Ambassador Guest Blog: The Secret to Navigating Life with a Rare Disease? Say Yes.
Charlotte is 11 years old and lives with LGMD2C, which was diagnosed when she was
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How art fosters a sense of belonging in my life with Duchenne
On Jan. 31, I visited the former Supreme Court Foyer at the National Gallery Singapore
