Category: Research

PTC today announced promising 12 month results from their Phase 2 PIVOT-HD study of oral PTC518  

At Month 12, mutant Huntingtin (mHTT) in the blood was lowered by 22% and 43% respectively for 5mg and 10mg doses. A similar result was seen in cerebrospinal fluid, where […]

Sage Therapeutics Phase 2 Study Reinforces Cognitive Impact of Huntington’s Disease

Earlier this week Sage Therapeutics announced the results of their SURVEYOR study which quantified the cognitive impact of Huntington’s Disease.  Using the HD-Cognitive Assessment Battery (HD-CAB) they measured the difference […]

FDA grants uniQure Regenerative Medicine Advance Therapy (RMAT) Designation

Today we are very happy in the Huntington community.  The announcement from Uniqure that the company has received the Regenerative Medicine Advanced Therapy (RMAT) “Designation for Investigational Gene Therapy AMT-130 […]

SOM Biotech completes the recruitment process in the Phase IIb study of the treatment of chorea in Huntington’s disease

SOM Biotech, a clinical-stage drug discovery and development company based on a unique proprietary Artificial Intelligence platform (SOMAIPRO®), is pleased to announce that the recruitment of the Phase IIb clinical […]

Prilenia Plans to Submit Marketing Authorization Application in the EU for Pridopidine in Huntington’s Disease

Prilenia announced today that they plan to submit a Marketing Authorization Application in the EU for the use of Pridopidine in the treatment of Huntington’s Disease. According to Dr. Michael […]

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 […]