We need your voice. Recent reporting raises concerns that the FDA may be changing expectations at the end of gene therapy trials, including the trial for the Huntington's disease (HD) treatment AMT-130 being developed by uniQure. For patients and families who are...

Patient advocacy plays a key role in bringing about systemic change in the development and approval of medical products. Ideally, patient engagement involves more than participation in clinical trials. It means active participation in providing input into...