XTalks

APRIL 15, 2025

In the pancreas, potassium channels regulate insulin secretion. Related: On Rare Disease Day 2025, We Need More Than You Can Imagine Today marks a historic day for the PWS community. Vykay XR Clinical Trial Results Data from a randomized, placebo-controlled Phase III trial (Study 2-RWP or Study C602-RWP) support Vykat XRs approval.

FDA Approval 52

FDA Approval Clinical Trials Clinical Trials Trials 52

XTalks

SEPTEMBER 14, 2022

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.

Life Science 90

Life Science FDA Approval Insulin Drugs 90

XTalks

SEPTEMBER 17, 2020

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. Also known as Berardinelli-Seip syndrome, there are four subtypes of lipodystrophy.

Trials 98

Trials Gene Hormones Clinical Trials 98