Author Archives: Patricia Inacio PhD

A Phase 1/2 clinical trial evaluating AMT-130 as a potential gene therapy for Huntington’s disease has “treated” its first two patients, uniQure announced. One person was given AMT-130 and the other a sham surgery as part of a control group. Both will now be followed for three months, after which a Data…

Vaccinex’s Phase 2 SIGNAL study, evaluating the investigational antibody pepinemab (VX15/2503) in Huntington’s disease patients with late prodromal and early disease, is ongoing and has tested pepinemab in all 265 patients enrolled in cohort B, the company announced. As of March 31, only 37 patients in this group…

Roche has completed patient enrollment for its Phase 3 GENERATION HD1 clinical trial evaluating the potential of tominersen to delay the progression of Huntington’s disease. A total of 791 adult patients across approximately 100 clinical sites around the world have been enrolled and the trial is expected to be…

A Phase 1 clinical trial testing Roche’s investigational therapy tominersen for Huntington’s disease has been placed on hold after two cases of infection were found during the study. The infections were linked to the device used to take samples of patients’ cerebrospinal fluid (CSF, the…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

New preclinical data continues to support uniQure’s AMT-130 investigational gene therapy for Huntington’s disease. Results reveal the therapy lowered the levels of toxic huntingtin protein in nerve cells derived from Huntington’s patients as well as in mouse, mini-pig and non-human primate models of the disease. The results were shared…

Rexulti (brexpiprazole), an antipsychotic medicine approved for schizophrenia and in some cases depression, eases motor and psychiatric symptoms of Huntington’s disease, according to a case study. The study, “Effectiveness of Brexpiprazole in the Treatment in a Patient with Huntington’s Disease,” was published in the…

AMT-130, uniQure’s experimental gene therapy candidate for Huntington’s disease, halts shrinkage of certain brain regions and helps preserve cognitive function in a mouse model of the disease, preclinical data show. Additionally, a single dose of AMT-130 reduced the levels of mutated huntingtin (HTT) — the protein that…

A small RNA molecule known as microRNA-1, found in both worm and human cells, can promote the removal of toxic protein aggregates — hallmarks of neurodegenerative diseases — by triggering a self-cleaning process called autophagy, a study shows. These findings may open new avenues for potential therapeutics for disorders such…

MC10 and the University of Vermont have teamed up to advance understanding of gait impairments in Huntington’s disease and other movement disorders. The partnership will combine MC10’s BioStamp nPoint device to measure physical parameters (biometrics) obtained from the lower limbs with the University of Vermont’s biomedical…