Category: Research

Breakthrough represents a new hope for HD patients

Scientists from the Weizmann Institute in Israel have published the results of an animal study which holds tremendous promise for the treatment of Huntington’s Disease.  They have discovered that two […]

uniQure Announces Update on Phase I/II Clinical Trials of AMT-130 Gene Therapy for the Treatment of Huntington’s Disease

~ Patients treated with AMT-130 continue to show evidence of preserved neurological function with potential dose-dependent clinical benefits relative to an inclusion criteria-matched natural history of the disease ~ ~ Mean […]

PTC Therapeutics Shares Positive Interim Data from PIVOT-HD Clinical Trial in Huntington’s Disease Patients

June 21, 2023 PDF Version – Dose-dependent lowering of blood Huntingtin (HTT) protein levels at 12 weeks –– Favorable tolerability profile with no treatment-related serious adverse events or NfL spikes –  – Conference […]

uniQure Announces Update on U.S. Phase I/II Clinical Trial of AMT-130 Gene Therapy for the Treatment of Huntington’s Disease

~ AMT-130 continues to be generally well-tolerated across both dose cohorts ~ ~ Patients treated with AMT-130 show preserved function compared to baseline and clinical benefits relative to natural history […]

PRILENIA ANNOUNCE RESULTS FROM THE PROOF-HD STUDY: PROMISING, BUT MIXED RESULTS!

Today, April 25th, Prilenia announced results from the PROOF-HD clinical trial for Huntington’s Disease. Some of the participants showed significant benefits from the Pridopidine drug, while others did not benefit at all.  This […]

Prilenia Achieves Last Patient Last Visit in Phase 3 PROOF-HD Clinical Study for Huntington’s Disease

PROOF-HD is the only late-stage study in Huntington’s disease targeting clinical progression; topline results expected in early Q2 2023 NAARDEN, Netherlands and WALTHAM, Mass., 28 March 2023 – Prilenia Therapeutics B.V., a […]

Roche/ Genentech update about GENERATION HD2 study

Roche/ Genentech announce that Phase II GENERATION HD2 study is now open. This is great news for the international community since it is planned to run in 15 countries (Argentina, […]

Novartis ends the VIBRANT-HD study

Novartis shared today the difficult news that they are stopping the VIBRANT-HD study of Branaplam, after some participants devoloping side effects. Read the full Novartis letter Even if this news […]

uniQure announced they are resuming the patient enrollment at the higher-dose level in AMT-130 trial

A Message to the Huntington’s Disease Community from uniQure Dear Huntington’s Disease Community Members, This morning, uniQure issued a press release which included an update on our clinical trial ofAMT-130 […]

Wave Life Sciences Announces Positive Update from Phase 1b/2a SELECT-HD Trial with Initial Results Indicating Allele-Selective Target Engagement with WVE-003 in Huntington’s Disease

September 20, 2022 at 7:30 AM EDT PDF Version Single doses of WVE-003 appear generally safe and well-tolerated CSF mutant huntingtin (mHTT) protein was reduced following single doses of 30 […]