News

PTC-AADC Gene Therapy’s European Approval Process Moves Forward

A European Medicines Agency committee is expected to provide an assessment of PTC-AADC (eladocagene exuparvovec) gene therapy for treating aromatic l-amino acid decarboxylase (AADC) deficiency this month, which will inform the final decision about whether the treatment will receive marketing approval in Europe. The therapy’s developer, PTC…

Organizations Rally to Help Ukrainian Rare Disease Patients

A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave. The treacherous, 34-hour pilgrimage that ultimately brought the group of eight by car to temporary accommodations in neighboring Poland last month was physically and emotionally difficult,…

Europe Awaits Proposed New Framework for Sharing Health Data

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

RTW Charitable Foundation to Fund Therapy Pipeline for Rare Diseases

The RTW Charitable Foundation will offer grants up to $150,000 per year to develop therapeutics for ultra-rare diseases, including aromatic l-amino acid decarboxylase (AADC) deficiency. The foundation, which supports research that may not have strong commercial backing, provides capital, human resources, and logistical support to help scientists accelerate…