Pfizer enrols first UK participant into global DMD gene therapy study
The CIFFREO study is expected to enrol 99 ambulatory male patients aged four to seven years old
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Tag: Duchenne muscular dystrophy
Lilly to collaborate with Precision BioSciences on genome editing research
Lead programme will focus on developing therapies for Duchenne muscular dystrophy
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Pfizer scores a speedy review in the US for Duchenne gene therapy
Fast track designation is based on promising phase Ib results
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EMA validates Santhera’s Puldysa MAA
Santhera expects an opinion by the CHMP around mid 2020.
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Santhera’s Syros succesful in DMD study
Santhera Pharmaceuticals has announced results from its Syros DMD study.
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Genome editing successful in correcting muscular dystrophy in mice
Researchers at Duke University have shown that CRISPR genome editing technology can safely and stably correct Duchenne muscular dystrophy in mice.
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Pfizer ends two Duchenne muscular dystrophy drug trials
Pfizer is shutting down two clinical studies assessing the anti-myostatin monoclonal antibody domagrozumab (PF-06252616) for the treatment of Duchenne muscular dystrophy (DMD).
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Early access to Santhera’s DMD therapy Raxone extended in the UK
UK regulators have renewed the scientific opinion for Santhera’s Raxone, extending its availability to patients with Duchenne muscular dystrophy (DMD) through the Early Access to Medicines Scheme (EAMS) for another year.
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Duchenne patients get NHS access to PTC’s Translarna
Cost regulators for the NHS in England and Wales have now issued final guidance recommending the use of PTC Therapeutics’ Translarna for patients with Duchenne muscular dystrophy.
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