Shionogi enrols first patients in Esprit trial for Pompe disease

The trial will examine the efficacy, safety, and pharmacodynamics of S-606001 SRT used with ERT in confirmed LOPD adults.
ascopubs.org

Malignant glioma subset from Actuate 1801: A phase 1/2 study of 9-ING-41, a glycogen synthase kinase 3 beta (GSK-3β) inhibitor, as a single agent and combined with ...

A phase I/IIa study to evaluate the safety and efficacy of blood-brain barrier (BBB) opening with the SonoCloud-9 implantable ultrasound device in recurrent glioblastoma patients receiving IV ...
ascopubs.org

Glycogen synthase kinase-3 beta (GSK-3β) blockade with 9-ing-41 in gastrointestinal cancers: The 1801 phase Ib/II study.

Clinical activity of avapritinib in ≥ fourth-line (4L+) and PDGFRA Exon 18 gastrointestinal stromal tumors (GIST). This is an ASCO Meeting Abstract from the 2020 Gastrointestinal Cancers Symposium.
The New England Journal of Medicine

Cardiomyopathy and Exercise Intolerance in Muscle Glycogen Storage Disease 0

Storage of glycogen is essential for glucose homeostasis and for energy supply during bursts of activity and sustained muscle work. We describe three siblings with profound muscle and heart glycogen ...
EurekAlert!

For mice, too much muscle glycogen impairs endurance exercise performance

In 2009, Usain Bolt set the world record in the 100-meter dash, thanks in large part to a carb called glycogen. This molecule is stored in skeletal muscle and later released to fuel short and intense ...