Surgery guided by MRI and computer navigation safely and accurately delivered Upstaza (eladocagene exuparvovec, marketed as Kebilidi in the…
Articles by Margarida Maia, PhD
Children with rare diseases who have a gastrostomy tube, a feeding tube inserted through the belly and into the stomach,…
A newly developed cell model has been called “a significant step toward understanding the molecular mechanisms of AADC deficiency”…
The Pennsylvania Senate has approved legislation to extend support for patients with rare diseases, such as aromatic L-amino acid…
A podcast from the Pacific Northwest Research Institute (PNRI) called PNRI Science: Rare Disease, Real Progress returns for its…
The European Rare Diseases Research Alliance (ERDERA) was launched officially last week with the vision to offer better diagnosis…
The Department of Health-Abu Dhabi and AstraZeneca are partnering to set up a center of excellence for rare diseases…
UCB has launched Aspire4Rare, a framework to “future-proof” health policies for rare diseases like aromatic l-amino-acid decarboxylase (AADC)…
PTC Therapeutics plans to seek the U.S. approval this month of Upstaza (eladocagene exuparvovec), its one-time gene therapy…
Measuring the levels of 3-O-methyldopa (3-OMD), a molecule that builds up in aromatic l-amino-acid decarboxylase (AADC) deficiency, can diagnose…