Rare disease drug developer BioMarin Pharmaceutical is buying its way to revenue growth with a $4.8 billion deal for Amicus Therapeutics, a company whose two commercialized products each offer ...
In early trial data, INZ-701 was associated with several clinical benefits including a reduction or stabilization of carotid intima-media thickness. The Food and Drug Administration (FDA) has granted ...
An ultra-rare enzyme deficiency that keeps infants from achieving proper muscle function among other developmental delays now has its first FDA-approved treatment. The new product, a gene therapy ...
Verywell Health on MSN
How serious is MCAD deficiency?
Facts About Medium-Chain Acyl-CoA Dehydrogenase Deficiency ...
BUFFALO, N.Y. – A University at Buffalo researcher working on Krabbe disease (KD) has been awarded a $250,000 grant from the Rosenau Family Research Foundation to investigate how an enzyme deficiency ...
AZoLifeSciences on MSN
DHPS enzyme deficiency impairs macrophage development and tissue repair
A new study led by researchers at the Johns Hopkins Kimmel Cancer Center, its Bloomberg~Kimmel Institute for Cancer ...
A UB researcher working on Krabbe disease (KD) has been awarded a $250,000 grant from the Rosenau Family Research Foundation to investigate how an enzyme deficiency in the brain causes this disease, ...
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