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FDA Approval of Sanofi’s Enzyme Replacement Drug for ASMD + Disney Actress Partners with Medtronic for Diabetes Tech Campaign – Xtalks Life Science Podcast Ep. 77

XTalks

In this episode, Ayesha discussed the FDA approval of Sanofi’s enzyme replacement therapy Xenpozyme for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD), a rare genetic lysosomal storage disease, in adults and pediatric patients.

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Surrogate Endpoints: How to Choose the Best One for Your Rare Disease Trial

XTalks

Acromegaly Surrogate Endpoint: Serum growth hormone and serum insulin-like growth factor 1 (IGF-1) are acceptable surrogate endpoints for acromegaly clinical trials involving somatostatin analogs such as octreotide, lanreotide and pasireotide. Novartis’ Signifor (pasireotide) is also indicated in the treatment of the disease.

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