Category: Research

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 […]

uniQure Announces Update on Low-Dose Cohort in Phase I/II Clinical Trial of AMT-130 Gene Therapy for the Treatment of Huntington’s Disease

~ Treatment generally well-tolerated with no significant safety issues related to AMT-130 in treated patients through one year of follow-up ~ ~ A mean reduction of 53.8% of mutant HTT […]