Gene therapy approved for Duchenne muscular dystrophy
The first approved gene therapy for certain paediatric Duchenne muscular dystrophy (DMD) patients leads to production of the Elevidys micro-dystrophin protein.
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The first approved gene therapy for certain paediatric Duchenne muscular dystrophy (DMD) patients leads to production of the Elevidys micro-dystrophin protein.
Ongoing clinical trials for oligonucleotide-based therapies will propel the oligonucleotide synthesis market to reach $16.7 billion by 2027, states a new report.
The pharmaceutical company has received a complete response letter for its golodirsen injection, which had been submitted for accelerated approval.