A Phase 1/2 clinical trial testing uniQure’s potential gene therapy AMT-130 in people with Huntington’s disease can continue enrollment, according…
Articles by Patricia Inácio, PhD
A Phase 3 clinical trial investigating the efficacy and safety of oral pridopidine for those with early stage Huntington’s…
The U.S. Food and Drug Administration (FDA) has granted an orphan drug designation to SOM3355, an investigational treatment for…
A Phase 1/2 trial evaluating Wave Life Science’s investigational therapy WVE-003 for Huntington’s disease has started dosing patients,…
A history of high alcohol intake seems to be linked to increased motor impairment and a higher burden of psychiatric…
Treatment with pridopidine rescues the function of mitochondria — the cell’s powerhouses — boosting energy production, and reduces oxidative stress…
Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will…
A Phase 1/2 trial testing the safety and efficacy of AMT-130…
uniQure has completed patient enrollment for the first group participating in a Phase 1/2 trial assessing the…
More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less…