Tag: Huntingtin Lowering

Results of Wave’s huntingtin lowering trial explained – video!

Just before the new year, Wave Life Sciences announced that the drug in their huntingtin lowering trial, PRECISION-HD2, had successfully lowered the concentration of mutant huntingtin protein, while healthy huntingtin was left unchanged. Video below! Why is this so exciting? To put it simply: if you inherit Huntington’s disease, you have one sick gene and one healthy gene. Both genes code for a…
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DAY 3: CHDI Therapeutics Conference

The 15th Annual Huntington’s Disease (HD) Therapeutics Conference was taking place in Palm Springs  from February 24 to 27. Here is a summary of the third day, put together by tweets from HDBuzz.  By HDBuzz and Phylis Kitema | Photo: HDBuzz Session: Huntingtin lowering The topic on the third day was Huntingtin lowering – one of the major avenues to Huntington’s…
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DAY 2: CHDI Therapeutics Conference

The 15th Annual Huntington’s Disease (HD) Therapeutics Conference is currently taking place in Palm Springs. Here is a summary of the second day, put together by tweets from HDBuzz.  By HDBuzz and Phylis Kitema | Photo: HDBuzz Session: Path to prevention Background: Enroll-HD, TRACK-HD & Self Enroll Enroll-HD collects data from Huntington’s disease (HD) patients whether they have symptoms…
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DAY 1: CHDI Therapeutics Conference

The 15th Annual Huntington’s Disease (HD) Therapeutics Conference is currently taking place in Palm Springs. Here is a summary of the first day, put together by tweets from HDBuzz. By HDBuzz and Phylis Kitema | Photo: HDBuzz Morning session: Genotype and Phenotype The first session at the 15th annual HD Therapeutics Conference was “Genotype and Phenotype”, where the…
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Left to right: Anne Lennon Bird, Svein Olaf Olsen and Astri Arnesen.

Unpacking Wave’s PRECISION-HD2 huntingtin-lowering trial announcement

Just before the new year, Wave announced that the drug in the PRECISION-HD2 trial had successfully lowered the concentration of mutant huntingtin in the spinal fluid. By Dr Jeff Carroll | Edited by Dr Ed WildPublished January 03, 2020 at www.HDBuzz.net DNA-based drugs called antisense oligonucleotides, or ASOs, are now in multiple clinical trials in Huntington’s disease, aiming to lower production…
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2019 ends with exciting results from Wave PRECISION-HD2 trial!

Today, December 30, Wave announced the first preliminary results from the their study PRECISION-HD2 . So far the results are very exciting and promising. It’s very encouraging to enter a new year with such exciting news from Wave. I look forward with optimism to the next steps in these trials. Astri Arnesen, President EHA In PRECISION-HD2…
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A letter from Roche

Roche Pharmaceuticals is currently working on their Huntingtin Lowering drug, RG6042 (formerly known as IONIS-HTTRx). In the letter Patient Partnership Director, Mai-Lise Nguyen, write about the company and their philosophy on working with the HD patient community. On behalf of the Roche HD team Nguyen writes: – Since taking over development of RG6042 from Ionis…
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Amazing news – drug seems to improve symptoms

Yesterday Ionis Pharmaceutical presented new data from their Huntingtin lowering trial, IONIS-HTTRx. The data demonstrates a relationship between reduction of the disease-causing  Huntingtin protein and improved symptoms for people with early stage Huntington’s Disease. After 3 months of treatment, the patients seems to benefit from the drug – now known as RG6042. This is very…
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