While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

Although ASOs targeting liver cells are in clinical use and highly effective, so far the only oligonucleotide therapies ...

Patients with Duchenne muscular dystrophy now have new hope with gene therapy. And for an Illinois family, it’s a double blessing. On their family farm in rural Illinois, the Flessner boys have ...

an investigational gene therapy designed to treat Duchenne muscular dystrophy (Duchenne). SGT-003 is a next-generation gene therapy candidate utilizing AAV-SLB101, a proprietary capsid developed ...

Dyne CEO Joshua Brumm said that this year the Phase 1/2 studies in myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy ... therapies based on antisense oligonucleotides, nucleic ...

Similar results were observed with an ASO targeting a different region of dystrophin that is currently being used in a phase II clinical trial for Duchenne muscular dystrophy. Furthermore ...

Also Read: Solid Biosciences Outpaces Competitors In Duchenne Gene Therapy Development Despite being one year older (average age 7.18 years) than those treated in Part 1 (average age 5.98 years ...

Patients with Duchenne muscular dystrophy now have new hope with gene therapy. And for an Illinois family, it’s a double blessing.