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

Duchenne muscular dystrophy, limb girdle muscular dystrophy R2, epilepsy and other inflammatory diseases. It is administered through subcutaneous, inhalation route of administration. It is formulated ...

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 ...

What Is Duchenne Muscular Dystrophy ... this has not yet been shown. The first gene therapy to be approved for DMD, delandistrogene moxeparvovec (Elevidys), is only prescribed to people with ...

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 ...