Personalized antisense oligonucleotides (ASOs) may accelerate studies on treating Duchenne muscular dystrophy (DMD).
Every year, tens of thousands of babies are born with enigmatic illnesses...
Bobcat mRNA works differently than exon skipping or AAV-microdystrophy mechanisms. This next-generation method could lead to low-cost drugs for Duchenne muscular dystrophy.
Many exon skipping...
Next generation exon skipping therapies for Duchenne Muscular Dystrophy (DMD) represent a promising area of research aimed at addressing the underlying genetic mutations that...
Insmed is getting ready to use its ground-breaking gene therapy, INS1201, to combat Duchenne muscular dystrophy. Insmed intends to start Phase I clinical trials...