Category: Research

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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Updates from CHDI Therapeutics Conference

Photo, left to right: Svein Olaf Olsen and Astri Arnesen at the CHDI Therapeutics Conference in 2016. The 15th Annual Huntington’s Disease (HD) Therapeutics Conference was taking place in Palm Springs. Over four days, from February 24 to 27, drug development was discussed. Summary: DAY 1 | DAY 2 | DAY 3 The CHDI (Cure Huntington’s Disease Initiative) Foundation conference is a forum for drug…
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Meeting on how to improve care in Europe

In January, patient advocates visited the European Reference Network for Rare Neurological Diseases. During the meeting, they discussed how to provide better care and improve knowledge of rare diseases. From www.ern-rnd.eu | Meeting video below The aims of European Reference Network for Rare Neurological Diseases (ERN- RND) are to address the unmet needs of more…
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“RARE/D”: A NEW RARE DISEASE PODCAST

RARE/D is a new podcast series that started on December 29. On the podcast, topics such as genome editing and health care are being discussed by a multidisciplinary team. The series are now available across many platforms including iTunes and Spotify. Find the episodes here. Behind the podcast series is the the Whitworth group consisting…
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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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Trial Finder Launched in Europe

This Christmas, we launched the first HD Trial Finder in Europe. HD Trial Finder is a platform developed to make it easier for you to find out more about ongoing Huntington’s disease (HD) trials and studies in Europe. Right now, Huntington’s disease trials and studies are taking place all across Europe: GENERATION HD1, ENROLL-HD, PRECISION-HD1,…
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First human gene therapy trial in Huntington’s disease

UniQure announces key details of its planned trial to assess the safety and ability of AMT-130 gene therapy to lower the problematic huntingtin protein using a ‘single-shot’ virus delivery system. Article byDr Anna Pfalzer published at HDBuzz.net The European Huntington Association met uniQure in September to talk about the gene therapy trial. Go to video interview…
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