News
Alexion Pharmaceuticals (ALXN) has been one of the greatest growth stories in the ultra-rare disease space over the past decade, with their blockbuster C5 inhibitor…
Allegheny Health Network (AHN) believe a novel multidisciplinary approach to patient care, together with emerging diagnostic and therapeutic advances, can help to…
The Society for Cardiovascular Magnetic Resonance (SCMR), a Society dedicated to improving cardiovascular health by advancing the field of cardiovascular magnetic…
The FDA announced that it has approved patisiran infusion for the treatment of patients with peripheral nerve disease caused by hereditary transthyretin-mediated…
A UCL-led study has confirmed that some vials of a hormone used in discontinued medical treatments contained seeds of a protein implicated in Alzheimer's disease,…
Tiny protein structures called amyloids are key to understanding certain devastating age-related diseases. Aggregates, or sticky clumped-up amyloids, form plaques…
Toxic protein assemblies, or "amyloids," long considered being key drivers in many neuromuscular diseases, also playing a beneficial role in the development of…
Rare Disease Day takes place on the last day of February each year. The main objective of Rare Disease Day is to raise awareness amongst the general public and…
Due to the global COVID-19 (Coronavirus) pandemic, the American Academy of Neurology has no choice but to make the necessary decision to cancel the AAN's Annual…
Pfizer Inc. announced today that the US Food and Drug Administration (FDA) accepted for filing the company's New Drug Applications (NDAs) for tafamidis for the…