Discover the importance of vision, genetic eye disorders, and RNA-based therapies for precision treatment in India and ...

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 ...

There is a simple discount patient access scheme for vamorolone. NHS organisations can get details on the Commercial Access and Pricing (CAP) Portal. Non-NHS organisations can contact ...

Current work is focussing on regulating splicing of the SMN2 gene in spinal muscular atrophy and antisense-oligonucleotide-mediated exon skipping in Duchenne Muscular Dystrophy. We are also ...

Some types of muscular dystrophy cause irregular heartbeats. Gene therapy. Delandistrogene moxeparvovec (Elevidys) is a gene therapy approved to treat Duchenne MD. It's only prescribed to people ...

Patients with spinal muscular atrophy (SMA) type 2 showed improved motor ability when treated with intrathecal onasemnogene abeparvovec, an investigational gene therapy. “Maintaining motor ...

PST-611-CT1 is a first-in-human single ascending dose trial aiming to confirm the favorable safety profile of the drug and validate the maximal dose for the Phase II proof-of-concept study, in six to ...

PST-611 is a first-in-class non-viral vectorized therapy for the treatment of dry ... s ability to protect retinal cells from atrophy and preserve vision.” Dirk Sauer, Chairman of the Board ...