Exercise has gotten a lot of media attention in the recent past as a way for people with neurological disorders to manage their symptoms and potentially change the progression of their disease. As part of our new web series, HDSA recently hosted an educational webinar on exercise and HD, led by Lori Quinn, a physical therapist, HD clinical researcher, and professor at Columbia University, and Dr. Nora Fritz, Assistant Professor of Physical Therapy at Wayne State University. They covered research into exercise and HD as well as guidelines for exercise and physical therapy for people with HD, and even led a demonstration of physical therapy exercises that can be modified for many levels. We encourage you to check out the recorded Exercise and HD webinar on HDSA’s youtube channel.
Continuing HD Education for Genetic Counselors
With so many genetic therapies in the HD research pipeline, an important consideration for the near future is how to meet an increased demand for HD genetic testing and ensure that families get expert advice and knowledgeable care. For this reason, HDSA and the National Society of Genetic Counselors (NSGC) have collaborated to provide a five part free online course for genetic counselors. The course aims to educate genetic counselors about the intricacies of Huntington’s disease so they are better able to apply their knowledge and skills about hereditary diseases when counseling a person at risk or a symptomatic individual. Professional education that includes an overview of the research landscape is one way that HDSA aims to increase national access to expert HD care.
Oral Genetic Therapy for Infants with Spinal Muscular Atrophy Shows Promise
Encouraging results were announced this week from a trial of risdiplam, a drug to treat a fatal inherited disorder that affects infants. Babies with spinal muscular atrophy (SMA) type 1 have progressive loss of the nerve cells in the spine that control movement, leading to muscle weakness. Many are never able to roll or crawl and eventually they are unable to swallow or breathe, dying as infants or during early childhood. SMA is caused by the loss of the SMN-1 protein. Risdiplam is a genetic drug taken by mouth that can boost the production of another protein to compensate for the loss of SMN-1.
The reason we’re excited about this news is that similar genetic drugs are being designed for HD: drugs that could be taken by mouth to lower the amount of huntingtin protein. Genentech and PTC Therapeutics, the companies behind risdiplam, are also working in the HD space, where they have therapies in the pipeline. Advancements in this area of medicine for one inherited disease are encouraging for other communities affected by genetic disorders, including HD. Check out HDSA’s HD research pipeline for more information about the development of genetic therapies and other treatments for HD.