Reviving the FDA

A recent article in the New England Journal of Medicine reviews current climate and barriers to change at the FDA with applicability to developing rare disease therapeutics.
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  • Congenital Muscular Dystrophy

    A group of diseases causing muscle weakness at birth. Several defined genetic mutations cause muscles to break down faster than they can repair or grow. A child with CMD may have various neurological or physical impairments. Some children never gain the ability to walk, while others lose the ability as they grow older. Learn more...

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