I'm dying of a fatal disease. Bureaucrats shouldn’t decide how doctors can try to save me
I'm fighting a fatal disease that took the life of my brother. I work in biotech and know there are medical advances that ...
'It's cruel that I cannot get muscular dystrophy drug'
A 12-year-old boy with a severe form of muscular dystrophy has said it is "cruel" that a new drug is not being made available ...
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What To Know About Duchenne Muscular Dystrophy (DMD)
Medically reviewed by Smita Patel, DO Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed ...
Massachusetts families, lawmakers fighting to keep NIH funding for life-saving medical research
Families and doctors spoke out against the cuts at a roundtable at the UMass Chan Medical School in Worcester, Massachusetts.
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Duchenne gene therapy from Solid Biosciences produces promising results in early study
Solid said that the first patients to receive its Duchenne gene therapy all produced high levels of the target protein, an ...
US health agencies postpone rare diseases event in an unusual move
A two-day meeting aimed at raising awareness among policymakers and public about rare diseases scheduled for the end of the ...
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Woman sees royal interest in her book, A Legacy of Love
Ann Blake, from Godshill, created her book, A Legacy of Love, which compiles poetry and artwork by several authors, including Jools Holland, ...
The Nolan Show - Young boy with severe form of muscular dystrophy says it is 'cruel' that a new drug is not being made available in NI - BBC Sounds
Young boy with severe form of muscular dystrophy says it is 'cruel' that a new drug is not being made available in NI ...
Entrada Therapeutics: Cash-Rich Innovator With Asymmetric Upside
Entrada Therapeutics' innovative Endosomal Escape Vehicle platform, poised to revolutionize drug delivery. Click here to read ...