NORWELL, Mass. — The National Organization for Rare Disorders (NORD®) today announced the addition of seven leading academic medical and research centers to its national NORD® Rare Disease Centers of Excellence Network, expanding collaboration to improve care and accelerate research for the more than 30 million Americans living with rare conditions. With...

By Raffaele Pereno – PhD, MBA VIENNA, Austria – A discussion with AATech CEO Rüdiger Jankowsky at BioEurope highlighted a decisive shift in how alpha‑1 antitrypsin (AAT) may be deployed in respiratory medicine. While most companies in the field continue to focus on treating the rare genetic disorder alpha‑1 antitrypsin...

New York, NY – Measuring the amount of glucosylsphingosine, also called lyso-Gb1, a key marker used to help diagnose Gaucher disease, may aid doctors in deciding when someone with Gaucher disease type 1 should begin treatment, a new study suggests. Additionally, a certain level of lyso-GL1 may help distinguish between...