Press Release - Axovant Gene Therapies Announces US FDA Clearance of IND for Registrational Study of AXO-AAV-GM2 Gene Therapy in Tay-Sachs and Sandhoff Diseases, allowing clinical trials in the United States.

Medication Survey in Patients with LOTS
Kyle Anthony, BAKyle BA
Susan HattersSusan Hatters--Friedman, M.D.
Jose Americo Fernandes Filho, M.D.
Barbara E. Shapiro, M.D.,Ph.D.
University Hospitals of Cleveland University Cleveland
Case Western Reserve University School of Medicine Case Medicine

Abstract—

The authors conducted a retrospective and brief prospective study of adverse effects of approximately 350 medications in 44 adults with late onset Tay–Sachs disease (LOTS). Some medications were relatively safe, whereas others, particularly haloperidol, risperidone, and chlorpromazine,
were associated with neurologic worsening.

2016 Request for Proposal announced.  Pre-applications due December 22.

NTSAD is soliciting proposals for innovative research projects that involve basic research, translational studies or clinical studies in the areas of neurodegenerative disorders affecting the CNS, including lysosomal storage disorders (particularly Tay-Sachs, GM-1, Sandhoff) and pediatric leukodystrophies (in particular Canavan disease).

A schedule for the NTSAD Science Symposium & Workshop to be held April 16, 2015 in Reston, Virginia at the Hyatt Regency hotel. 

The Lysosomal Disease Network accepting applications until Friday, February 6th, 5:00 PM (CST).

Updated guidelines can be found here with new deadlines.