The trial will examine the efficacy, safety, and pharmacodynamics of S-606001 SRT used with ERT in confirmed LOPD adults.
S-606001 has the potential to become the first oral substrate reduction therapy for Pompe diseaseOSAKA, Japan--(BUSINESS WIRE)--Shionogi & Co., Ltd. (Head Office: Osaka, Japan; Chief Executive Officer ...
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.