News

Two studies from the lab of Daryl Bosco, PhD, offer new insights into the biochemical and molecular mechanisms that cause amyotrophic lateral sclerosis.

Five runners are participating in the 125th running of the Boston Marathon on Oct. 11 to raise money for UMass Medical School’s ALS Cellucci Fund.

The Dan and Diane Riccio Fund for Neuroscience has awarded four teams of UMass Medical School researchers $50,000 seed grants for interdisciplinary collaborations leading to innovative discoveries that deepen understanding of normal brain function and what goes awry in neurological diseases.

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.

Thomas Feldborg and Daria Rokina, of Denmark, chose to enroll their baby daughter, Alissa, in an investigational gene therapy for the treatment of GM2 gangliosidosis, which causes Tay-Sachs and Sandhoff diseases, to “let hope conquer the fears,” according to a report in USA Today chronicling their journey.

Apic Bio, a gene therapy company developing treatment options for patients with rare genetic diseases and co-founded by UMass Medical School’s Robert H. Brown Jr., DPhil, MD, announced that the FDA has cleared its investigational new drug application for APB-102, a gene therapy candidate designed to treat a common cause of familial amyotrophic lateral sclerosis.

The annual event, designed to stimulate high school students’ interest in neuroscience, was hosted by the Departments of Psychiatry and Neurology and the NeuroNexus Institute at UMass Medical School via Zoom on Saturday, March 20.

The 15th annual Brain Bee competition for Massachusetts high school students will be hosted by the Departments of Psychiatry and Neurology and the NeuroNexus Institute at UMass Medical School via Zoom.

Oguz Cataltepe, MD, describes the innovative neurosurgical procedure used to treat patients in the new clinical trial for a gene therapy targeting infantile Tay-Sachs and Sandhoff diseases.

Sio Gene Therapies announced that the first patient with infantile Tay-Sachs disease has been dosed in a Phase I/II trial evaluating AXO-AAV-GM2, an investigational gene therapy for the treatment of GM2 gangliosidosis, which causes Tay-Sachs and Sandhoff diseases. Miguel Sena-Esteves, PhD, is a principal scientist of the research program at UMMS.

Axovant Gene Therapies, a clinical-stage company developing innovative gene therapies based on UMass Medical School research discoveries, has announced that the FDA has lifted its clinical hold and cleared Axovant’s Investigational New Drug application to initiate a registrational study of gene therapy to treat patients with Tay-Sachs disease and Sandhoff disease.

On behalf of the UMass Medical School community, we extend our sincere condolences to Jake Kennedy’s family and to all who knew and loved him. Jake Kennedy died Tuesday, Oct. 13, at age 65, in his Salem, N.H., home of amyotrophic lateral sclerosis.

UMass Medical School and Massachusetts General Hospital are the first to safely treat two research participants with a synthetic microRNA, delivered into the spinal fluid, designed to silence a human disease-causing gene. Details of the treatment appear in the New England Journal of Medicine.

Salome Funes, PhD candidate in the Graduate School of Biomedical Sciences, and the Society for the Advancement of Chicanos and Native Americans in Science are reaching out to organizations like Girls Inc. to teach young women about careers in science.

In a new Voices of UMassMed podcast, Kara Smith, MD, talks about early warning signs of Parkinson’s disease and new developments in research.

The Angel Fund for ALS Research donated $1 million last year to support the research of Robert H. Brown Jr., DPhil, MD. The sole mission of the nonprofit organization is to find a treatment and cure for ALS.