A less invasive airway device — called a supraglottic airway device or SAD — was found to safely and effectively…
Articles by Marta Figueiredo, PhD
The gene therapy Upstaza (eladocagene exuparvovec), formerly PTC-AADC, has been recommended for approval in the European Union for patients,…
Centogene has launched CentoCloud, a cloud-based platform designed to allow decentralized analysis and interpretation of an individual’s genetic…
Researchers have developed and validated new tools to help evaluate health-related quality of life, or HRQoL, in people with…
Delivering the investigational gene therapy PTC-AADC to a specific part of the brain, called the putamen, was found to…
Caregivers of children with aromatic l-amino acid decarboxylase (AADC) deficiency spend the bulk of their days providing care, with most…
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of…
PTC Therapeutics has developed and validated tools to help assess health-related quality of life (HRQoL) in people with…
The first wave of COVID-19Â in Europe severely disrupted access to care and raised stress and anxiety in people with…
PTC Therapeutics’ investigational gene therapy PTC-AADC is expected to result in long-term and durable therapeutic benefits in children with…