Nasdaq

REGENXBIO Announces Continued Progress and Expansion of Clinical Development Program for RGX-121 for the Treatment of Mucopolysaccharidosis Type II (MPS II)

- Ongoing Phase I/II trial to enroll up to 6 additional patients in Cohort 2 while planning for next steps in program; further trial updates expected by the end of 2020 - New IND for Phase I/II trial ...
Business Wire

MEDIPAL and JCR Announce Completion of Clinical Trial Notification Process in Japan for Phase I/II Study of JR-446 for Mucopolysaccharidosis Type IIIB

HYOGO, Japan--(BUSINESS WIRE)--MEDIPAL HOLDINGS CORPORATION (TSE 7459 “MEDIPAL”) and JCR Pharmaceuticals Co., Ltd. (TSE 4552 “JCR”) today announced the completion of the regulatory review by the ...
Nasdaq

Orchard Therapeutics Announces Interim Data for OTL-203 Showing Positive Clinical Results in Multiple Disease Manifestations of Mucopolysaccharidosis Type I Hurler Syndrome ...

IR webinar today at 4:30 p.m. ET with Dr. Simon Jones from Manchester Centre for Genomic Medicine to review OTL-203 and OTL-201 data BOSTON and LONDON, Feb. 09, 2021 (GLOBE NEWSWIRE) -- Orchard ...