...approval.PTC Therapeutics Inc. (NASDAQ:PTCT) reported Thursday after market close that Translarna ataluren failed to raise dystrophin... ...20 patients ages 2-7 in the intent-to-treat population of Study 045. On the primary endpoint, dystrophin... ...to present the results, CEO Stuart Peltz pointed out that after excluding two non-evaluable patients, IHC-measured dystrophin...
...41 patients with Duchenne muscular dystrophy. The study did meet the primary biological endpoint of micro-dystrophin... ...also will cut headcount by 20%.TARGETSBtk – Bruton’s tyrosine kinaseSGLT2 – Sodium-glucose cotransporter 2
RG6356 (SRP-9001, rAAVrh74.MHCK7.micro-Dystrophin)
...study of SRP-9001; Roche (SIX:ROG; OTCQX:RHHBY) has its ex-U.S. rights (see “Deal Could Extend Beyond Micro-Dystrophin”... ...its market cap is above $12 billion. Targets SGCB - sarcoglycan β
Paul Bonanos, Associate Editor