...Emflaza deflazacort from PTC Therapeutics Inc. (NASDAQ:PTCT) is also approved for the disease; PTC’s Translarna ataluren...

...is in development for Huntington disease. PTC markets four drugs: Duchenne muscular dystrophy therapies Translarna ataluren...

...designed to read through premature stop codons such as Duchenne muscular dystrophy (DMD) drug Translarna ataluren...

...myelogenous leukemia, and the upholding of its June recommendation against expanding the label of Translarna ataluren...
...Editor VSV-EBOV (V920, BPSC1001, rVSV-EBOV, rVSV-ZEBOV-GP, VSV-ZEBOV) Spravato, esketamine (jnj-54135419) Evenity, romosozumab (AMG 785, CDP7851, anti-sclerostin antibody) Translarna, ataluren (ptc124) Merck...

...care steroids; exon-skipping therapy Exondys 51 from Sarepta Therapeutics (NASDAQ:SRPT); and outside the U.S., Translarna ataluren...

...Euronext:UCB) to treat severe osteoporosis in postmenopausal women; and a label extension application for Translarna ataluren...
...5G1.1) Tecentriq (Brand), RG7446 (Compound #), MPDL3280A (Compound #), atezolizumab (Generic), Tecentriq (Other), Anti-PDL1 (Other) Translarna, ataluren (ptc124) Trogarzo...

...Johnson & Johnson (NYSE:JNJ), and issued a negative opinion regarding a label expansion for Translarna ataluren...
...MCL); and as monotherapy or in combinations for first- or second-line chronic lymphocytic leukemia (CLL). Translarna...
...#), ibrutinib (Generic), Imbruvica (Other) NutreStore (Brand), Endari (Brand), L-glutamine (Generic), Levoglutamide (Informal), Xyndari (Informal) Translarna, ataluren (ptc124) AbbVie...

...that otherwise likely would have been rejected. In 2016, NICE recommended the use of Translarna ataluren...
...of a managed access agreement. NICE did not estimate an incremental cost-effectiveness ratio (ICER) for Translarna...
...data. NHS England can stop funding treatment if the data do not support continued use. Translarna’s...

...FDA appeal request from PTC Therapeutics Inc. (NASDAQ:PTCT) regarding a complete response letter for Translarna ataluren...

...of children with Duchenne muscular dystrophy (DMD) during NICE’s health technology assessment (HTA) of Translarna ataluren...
...the time it took for them to discuss how to get it reimbursed,” Crossley said. Translarna...
...BioCentury. Godfrey was associate director of NICE’s Highly Specialised Technologies (HST) program during part of Translarna’s...