The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However,…
Articles by Patricia Inácio, PhD
New preclinical data continues to support uniQure’s AMT-130 investigational gene therapy for Huntington’s disease. Results reveal the therapy lowered…
Rexulti (brexpiprazole), an antipsychotic medicine approved for schizophrenia and in some cases depression, eases motor and…
AMT-130, uniQure’s experimental gene therapy candidate for Huntington’s disease, halts shrinkage of certain brain regions and helps…
A small RNA molecule known as microRNA-1, found in both worm and human cells, can promote the removal of toxic…
MC10 and the University of Vermont have teamed up to advance understanding of gait impairments in Huntington’s…
Increasing the levels of a natural enzyme called nicotinamide mononucleotide adenylyltransferase — or Nmnat — halts the formation of…
EIP Pharma is launching a proof-of-concept Phase 2 clinical trial to assess the ability of therapeutic candidate neflamapimod…
Emerald Health Pharmaceuticals announced that two of its cannabinoid-derived candidates — CBGA-Q and CBGA-Q-Na Salt — showed anti-inflammatory and…
RG6042, formerly called IONIS-HTTRx, a potential therapy for Huntington’s disease, was able to reduce the levels of mutant…