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BRIDGE grants support research into rare Sialidosis disease and patch to prevent pressure ulcers

Heather Gray-Edwards, PhD, DVM; Miguel Sena-Esteves, PhD; and Raymond Dunn, MD; are the recipients of the Office of Business Development & Innovation 2021 BRIDGE Fund grants.

The State of Gene Therapy

The State of Gene Therapy May 4, 2021 Time: 11:00 am PT, 2:00 pm ET Special Guests: James Wilson, MD, PhD (Perelman School of Medicine, University of Pennsylvania) Artur Padzik, PhD (AAV Production Manager, Biovian) Gene therapy has made tremendous strides over the past decade or so. The first approved drugs are on the market, and literally hundreds of trials are underway offering unprecedented hope to patients with rare, debilitating genetic disorders. Nevertheless, safety issues continue to swirl around the field as adverse events are reported in trials involving both lentivirus and AAV vectors. Pricing and manufacturing pose additional hurdles that could significantly challenge the field.

USA Today reports on first baby in gene therapy clinical trial for Sandhoff, Tay-Sachs diseases

Innovative neurosurgical technique used in Tay-Sachs gene therapy clinical trial

Innovative neurosurgical technique used in Tay-Sachs gene therapy clinical trial By Susan E.W. Spencer March 03, 2021 When the first patient in a new clinical trial was given a gene therapy targeting infantile Tay-Sachs and Sandhoff diseases in January, an innovative procedure that represents a “new frontier for neurosurgery” was used to deliver the therapeutic. Red arrows indicate the location in the thalamus where the infusion is targeted. The trial, by Sio Gene Therapies (formerly known as Axovant), is an open-label, two-stage, clinical study designed to evaluate safety and dose-escalation and safety and efficacy of surgical delivery of AXO-AAV-GM2 to the brain and spinal cord of trial participants with infantile or juvenile GM2 gangliosidosis. Sio licensed exclusive worldwide rights from UMass Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including

First patient dosed in clinical trial of gene therapy for Tay-Sachs and Sandhoff disease