...debate; with approvals based on the biomarker dystrophin...

...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...

...although SRP-9001 met the biological endpoint of micro-dystrophin...

...of a Phase III study of its micro-dystrophin...
...met the primary biological endpoint of increasing micro-dystrophin...
...vary, but data from animal models suggest micro-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 Stephen Hansen RG6356 (SRP-9001, rAAVrh74.MHCK7.micro-Dystrophin) InnoCare...

...SRP-5051 to treat DMD patients amenable to exon 51 skipping showed higher tissue exposure and dystrophin...

...Exondys approval on the surrogate endpoint of dystrophin...

...in the EU to treat nonsense mutation DMD.Sarepta is also developing a gene therapy targeting micro-dystrophin...

...In the case of muscular dystrophy, missing dystrophin...
...Asset Management Schroder Adveq Calporta Therapeutics Inc. Mucolipin 1 (TRPML1) (MCOLN1) Dystrophin Myeloid...

...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 MYO-101 SRP-9001, rAAVrh74.MHCK7.micro-Dystrophin Sarepta...