Over the past 15-20 years we have learned that the CAG repeat mutation that causes HD is dynamic – the DNA code isn’t constant, as expected, but tends to expand. This can happen from parent to child (sometimes causing juvenile HD). It can also happen throughout a person’s life in certain cells and organs, like…

HDSA Centers of Excellence provide world-class multidisciplinary care to families all over the United States. Professionals at these locations include neurologists, psychiatrists, social workers, therapists, and counselors with extensive HD experience who work together to provide care and support for families. Because HDSA is highly committed to supporting clinical research, Centers of Excellence must also…

This week, Ionis Pharmaceuticals issued a press release stating that the Phase 3 trial of the huntingtin-lowering therapy formerly known as IONIS-HTTRx has formally begun. This trial is in the hands of a larger company called Roche/Genentech, and the drug is now called RG6042. Roche/Genentech issued a letter to the global HD community which we…

This week, we learned that the SIGNAL trial has completed recruitment for its Phase 2 study of VX15/2503 (pepinemab), a drug made by Vaccinex. VX15 is an antibody designed to bind to the semaphorin 4D (SEMA4D) protein, a molecule that guides the activation and movement of cells within the body, and which may be responsible…

If you weren’t able to join us for the 2018 HD Research Year-in-Review webinar or would like to have another look, it’s now available on our YouTube channel. On Wednesday January 16th, along with HDSA’s Dr. George Yohrling, we presented an overview of HDSA’s research activities and hot topics in HD for 2018, talked about…

The American Society of Human Genetics (ASHG) is a nonprofit organization whose mission is to advance human genetics in science, health, and society through excellence in research, education, and advocacy. They hold a yearly essay contest for high schoolers, grades 9-12, promoting education and reflection upon genetic topics and awareness of DNA Day (Thursday, April…

To complement our 2018 Research Report, George and Leora will be sharing this year’s HD research highlights in our 2018 Research Year in Review Webinar on Wednesday, January 16th, 2019, from 12-1 pm Eastern time. Register to attend the webinar here.   New Year, New SAB Member HDSA’s Scientific Advisory Board (SAB) is composed of…

On Wednesday, December 19th, Roche/Genentech announced the US and Canadian locations for their upcoming study to test efficacy of the huntingtin-lowering therapy RG6042. This will be a 2-year study in people with early diagnosed HD. It will involve monthly lumbar punctures (spinal injections) to deliver RG6042 or a placebo, as well as clinical tests and scans…

This week, HDSA published the 2018 Research Report, a written re-cap of global HD research progress and how HDSA research dollars were put to use this year. We cover the work of HDSA-supported fellows, this year’s hot topics, conferences, clinical research news, and more, in a redesigned format. It’s a great summary of what happened…

The Ionis/Roche/Genentech Huntington’s program has received a lot of press in recent months. However, several additional companies have been working towards the same goal of developing therapies to target the genetic source of HD. One company is testing an antisense oligonucleotide (ASO) therapy, already in clinical trials, while others have novel drugs in the works…