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 59

FDA Approval Clinical Trials Clinical Trials Trials 59

XTalks

SEPTEMBER 17, 2020

In rare disease trials, it’s not always feasible to choose clinically-relevant endpoints to measure the efficacy of a new therapeutic. Verifying the biomarker’s clinical validity for use as a surrogate endpoint in rare disease research is another hurdle which is generally a longer-term goal.

Trials 98

Trials Gene Hormones Clinical Trials 98

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

pharmaphorum

AUGUST 24, 2021

It seems likely that the type 1 diabetes programme will be focused on developing cells that can secrete insulin in response to blood glucose levels – acting as a replacement for pancreatic beta cells that are destroyed in the autoimmune disease – which is already a focus of Vertex’ research.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52