Patricia Inácio, PhD,  science writer—

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…

Increasing the levels of a natural enzyme called nicotinamide mononucleotide adenylyltransferase — or Nmnat — halts the formation of mutant huntingtin (Htt) protein aggregates that build up in nerves cells in Huntington’s disease, a fruit fly study shows. “We discovered the neuroprotective role of a cellular ‘housekeeping’…

EIP Pharma is launching a proof-of-concept Phase 2 clinical trial to assess the ability of therapeutic candidate neflamapimod to treat cognitive dysfunction in patients with early-stage Huntington’s disease. The trial (NCT03980938), which is currently recruiting, is taking place at the John Van Geest Centre for…

Emerald Health Pharmaceuticals announced that two of its cannabinoid-derived candidates — CBGA-Q and CBGA-Q-Na Salt — showed anti-inflammatory and neuroprotective effects in a mouse model of Huntington’s disease. However, these benefits were not superior to those obtained from treatment with the company’s already patented EHP-102 compound. The…

RG6042, formerly called IONIS-HTTRx, a potential therapy for Huntington’s disease, was able to reduce the levels of mutant huntingtin (mHTT) protein in early-stage patients, according to results from a Phase 1/2 clinical trial. Trial results were published in the study, “Targeting Huntingtin Expression in Patients with…