This Week in HD Research: Discovery of the HD Gene and HD Gratitude Day  On March 26th, 1993, the discovery... The post This Week in HD Research, Survey Opportunities first appeared on Huntington's Disease Society of America ..read more

Clinical trial Participation and Disability   To file a disability claim, medical records are a must. But do clinical trial... The post Clinical trials and Disability, POWERHD, Online Survey first appeared on Huntington's Disease Society of America ..read more

2024 HDF Milton Wexler Biennial Symposium   Last week, HDSA staff were excited to join the Hereditary Disease Foundation’s Milton... The post Research Updates, Make-A-Will, Career Milestones first appeared on Huntington's Disease Society of America ..read more

Online Survey: Supporting Innovation in HD Drug Development   Provide the Food and Drug Administration, researchers, medical product developers and... The post Support Innovation in HD Drug Development, HD History  first appeared on Huntington's Disease Society of America ..read more

HDSA Announces 2024 Research Fellowship Recipients   Last week, HDSA announced the newest recipients of the Berman-Topper Family HD Career Development Fellowship, Dr. Thai Bao Nguyen from the University of California, Irvine, and Dr. Sonia Vázquez-Sánchez from the University of California, San Diego. This program seeks to  develop the next generation of passionate HD scientists by supporting innovative science conducted by early post-doctoral researchers with a desire to make HD part of their career plan. To learn more about the HDSA Berman-Topper HD Career Development fellowship and ..read more

Wave Life Sciences shares SELECT-HD Update  Earlier this week, Wave Life Sciences shared the results of SELECT-HD, a small phase 1b/2a clinical trial that tested the safety and efficacy of WVE-003. This spinally-delivered ASO drug targets a specific code, SNP3, that is present only on the allele containing the expanded copy of the huntingtin gene, which allows it to lower only the harmful form of huntingtin protein while preserving healthy huntingtin in people with HD. About 40% of people with HD have SNP3, so prospective participants had to take an additional genetic test to confirm thei ..read more

PTC Therapeutics shares update on PTC-518 PTC Therapeutics announced today results from PIVOT-HD, the Phase 2 study of PTC518 for HD. This experimental huntingtin-lowering therapy is a pill taken by mouth and is a splicing modulator, meaning that PTC518 interferes with and prevents huntingtin protein production by inserting a premature stop sign into the body’s message to make huntingtin protein. Results from 32 study participants who have completed 12 months of treatment with PTC518 showed dose-dependent lowering of expanded huntingtin protein in both blood and cerebrospinal fluid. This ..read more

Sage Therapeutics announces SURVEYOR update   Earlier this week, Sage Therapeutics announced the results of the SURVEYOR trial of a drug called dalzanemdor, previously known as SAGE-718, designed to treat cognitive symptoms of brain diseases.  The study met its key goals, confirming the impact of cognitive changes in HD using a test called the HD Cognitive Assessment Battery (HD-CAB). It also showed that a drug aimed at cognitive changes, called dalzanemdor, was safe in people with HD. Sage is performing further analysis to understand the relationship between changes in cognition and ..read more

HDSA’s 39th Annual Convention  Last week, HDSA welcomed over 750 members of the HD community to Spokane, WA for its 39th Annual Convention. The three days of learning and support around dozens of topics relevant to HD families included discussions on gene therapy, use of human tissue in HD research, HDSA-supported science, a Year in Review presentation from the HDBuzz team, and opportunities to Ask the Scientists Anything. Links to recorded sessions will be posted later this summer—stay tuned for more details in the near future!   uniQure granted RMAT designation for AMT-130&nbs ..read more

This Week in Research History: International Clinical Trials Day May 20th is recognized as Clinical Trials Day to commemorate what is regarded as the first randomized clinical trial initiated by James Lind on May 20, 1747. In this momentous study, Lind analyzed possible treatments of scurvy in shipmates aboard the HMS Salisbury of the British Royal Navy. As we commemorate 276 years of clinical trials and the vast advances in medical treatment and science that they brought about, HDSA extends its gratitude to clinical researchers, volunteers, and trial participants who allow us to press on in t ..read more