Drew Sinha
@genessohotrightnow.bsky.social
Happy to be on Bluesky and see so much work hosted from our department (including our paper)!
Case study in siblings with #ASMD shows earlier initiation of olipudase alfa led to better outcomes in ILD, organ size, and growth. Findings highlight the cumulative benefits of early ERT & support early diagnosis & intervention in pediatric patients. www.sciencedirect.com/science/arti...
Benefits of early intervention with olipudase alfa in symptomatic children with acid sphingomyelinase deficiency: A sibling case-comparison study
Acid sphingomyelinase deficiency (ASMD) is a lysosomal storage disease with multisystem complications including neurodegeneration, hepatosplenomegaly,…
www.sciencedirect.com
May 4, 2025 at 4:53 PM
Happy to be on Bluesky and see so much work hosted from our department (including our paper)!