News

INT41 Gene Therapy for Neurodegenerative Diseases Granted Priority Patent Status

The patent application for gene therapy candidate INT41, developed by Vybion for the treatment of Huntington’s disease, spinal muscular atrophy (SMA), and other neurodegenerative diseases, was granted Track One status from the U.S. Patent and Trademark Office (USPTO). The patent application covers the treatment’s composition and methods of use.

RTI to Offer Free Genetic-disease Tests for North Carolina Newborns

RTI International has developed a groundbreaking program of free genetic-disease testing for newborns, with the initial screenings being for the spinal muscular atrophy (SMA) and fragile X syndrome. Although most American newborns are tested for a number of genetic diseases, screenings are not done for others because they are too…

JPND Launches EU Request for Neurodegenerative Disease Research Projects

The EU Joint Programme —Neurodegenerative Disease Research (JPND) is calling for proposals for pathway analysis of multinational research projects, focusing on neurodegenerative diseases such as spinal muscular atrophy (SMA). According to a JPND news piece, neurodegenerative disorders such as Alzheimer’s disease and other dementias and Parkinson’s disease are…

Proof-of-Concept Study May Have Found New Way of Treating SMA

Researchers may have discovered a new treatment for spinal muscular atrophy (SMA). Identifying a molecule that prevents the SMN2 gene from being read by protein-making machinery, they then showed that getting rid of it increased SMN protein levels. Importantly, the approach also enhanced the effectiveness of a splice-modification drug that works much like the…