Novartis plans to start in-human Huntington’s disease trial in 2021

The Swiss pharmaceutical company Novartis is currently developing a pill for Huntington’s disease. Two days ago, the US Food and Drug Administration, FDA, granted an Orphan Drug Designation for the drug.  The drug is called branaplam (LMI070) and is administered in the form of a pill that you take through your mouth.  At the moment, […]

Two new participants have enrolled in gene therapy trial

In June, two people enrolled in the first gene therapy trial for Huntington’s disease. Now, two more participants have joined the study uniQure states in a new press release.  UniQure is currently developing a gene therapy for Huntington’s disease. The drug candidate is called AMT-130 and is a gene therapy. In other words, it seeks to […]

Targeting Unstable Genes

Due to instability in the gene, Huntington’s disease symptoms may begin earlier in life. By targeting proteins involved in DNA damage repair, we might be able to reduce the levels of instability and delay disease progression. Many repair proteins work to maintain the machinery that copies DNA.  One of these proteins – so-called Huntington’s disease […]

EHDN Newsletter: A special pandemic issue

The European Huntington’s Disease Network (EHDN) Newsletter appears three times a year. This year’s second edition is dedicated to the impact of Covid-19: the problems it has created but also the adaptability and the positive trends. In the newsletter, you can learn more about telemedicine. Here, Alzbeta Mühlbäck, our own doctor, states: – Though we […]

Update from Wave: Webcast and Ongoing Huntington’s Disease Trials

On Tuesday 25 August, Wave Life Sciences is organising a webcast where they are planning to share exciting updates on their platform. In a recent press release, the company also announced that the results of their huntingtin lowering trials are expected in the first quarter of 2021. 10 August 2020, Wave Life Sciences issued a […]

Join EHDN Virtual Bridging Event!

September 11 from 14:00 – 18:00 CEST, the European Huntington’s Disease Network will host Virtual Bridging Event where you can learn more about Huntington’s disease research and trials. Sign up now! Every other year, the European Huntington’s Disease Network (EHDN) hosts one of the world’s largest conferences dedicated solely to Huntington’s disease (HD). Despite taking a different form […]

Latest Huntington’s disease news!

The EHDN Newsletter is here! The 39th edition is dedicated to ethics in Huntington’s disease. Find it here. The European Huntington’s Disease (EHDN) newsletter aims to communicate the network’s activities and other developments of interest in the field of Huntington’s disease to the lay community, healthcare professionals and scientists. It appears three times a year (March 1, July […]

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 […]

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 […]