A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…
The U.S. Food and Drug Administration (FDA) is putting on hold Voyager Therapeutics’ request to test VY-HTT01, its investigational gene therapy for Huntington’s disease, in a clinical trial. The clinical hold will be kept in place until certain issues with VY-HTT01’s chemistry, manufacturing, and controls are…
I have decided to get my wife a T-shirt that reads, “It’s not you, it’s me.” I want to get it for her so she can wear it each time she visits a doctor. As if her having Huntington’s disease is not bad enough, eight months ago she started…
As I mentioned in my debut column of “Negative to Positives” a couple weeks ago, I have a genuine passion for helping those undergoing genetic testing for Huntington’s disease, which is an incredibly challenging life hurdle. Many members of this community watch their parents slowly and progressively decline, and…
No significant safety concerns associated with AMT-130, uniQure’s experimental gene therapy for Huntington’s disease, were identified in the first two patients participating in a Phase 1/2 trial assessing the treatment’s safety, tolerability, and efficacy. These were the main conclusions that came out of the first meeting of an…
I’m protective, a trait typical of most men. For example, I will drive for 18 hours across the country while my wife sleeps in the passenger seat. Jill has trouble sleeping in general, and the motion of the car acts as a sedative, rocking her to sleep. Long…
Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions…
When I first understood that I was going to be a writer, I did what everyone does upon discovering a passion: I wrote and wrote and wrote. I realized that the best writing gushes with precise details. That quality of writing required a lot of practice. So, I kept a…
Vaccinex’s investigational antibody pepinemab showed good tolerability in a Phase 2 clinical trial of people with late prodromal (before clinical diagnosis) and early Huntington’s disease (HD), top-line data show. The results also hint the treatment may improve cognition; however, those efficacy results did not reach statistical significance.
On Saturday, the last season of the Netflix series “The Good Place” began streaming. To me, it was the best ending of any TV show I have ever seen. My boyfriend and I cried. I’ll try to avoid spoilers, because…
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