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 CSF NfL continue to demonstrate favorable trends with low-dose patients below baseline at 30 months and high-dose patients near baseline Read more…

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 call and webcast to be held June 21st at 8:00 am EDT –  SOUTH PLAINFIELD, N.J., June 21, 2023 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today shared Read more…

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 of the disease ~ ~ Neurofilament Light Chain (NfL) in cerebrospinal fluid (CSF) was below baseline at 24 months in Read more…

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 is promising because for the first time we have a drug that demonstrates effect on disease progression, cognitive and motor Read more…

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 clinical stage biotechnology company focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases Read more…

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 (the name of the study drug) for Huntington’s Disease (HD.) After a brief pauseannounced this past August in patient enrollment Read more…

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 or 60 mg; mean mHTT reduction across both cohorts was 22% (median reduction 30%) from baseline 85 days post-single dose Read more…

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 (mHTT) observed in cerebral spinal fluid (CSF) at 12 months in evaluable patients treated with AMT-130 ~ ~ Neurofilament Light Read more…

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