Tag Archives: Rare

Researchers find immune component to rare neurodegenerative disease

UT Southwestern researchers have identified an immune protein tied to the rare neurodegenerative condition known as Niemann-Pick disease type C. The finding, made in mouse models and published online in Nature, could offer a powerful new therapeutic target for Niemann-Pick disease type C, a condition that was identified more than a century ago but still… Read More »

Albireo’s rare liver disease drug Bylvay scores back-to-back approvals in U.S., Europe

With a pair of approvals on both sides of the Atlantic, liver disease-focused biotech Albireo is off and running with its new medicine Bylvay. The oral drug, the first treatment for pruritus in patients with progressive familial intrahepatic cholestasis (PFIC), scored an FDA nod Tuesday and a European approval Monday. With the FDA’s blessing, Albireo is kicking off an “immediate”… Read More »

NIH Looking Into Rare Allergic Reactions to COVID Vaccines

THURSDAY, April 8, 2021 (HealthDay News) — A new clinical trial will investigate whether people who are highly allergic or have what’s known as a mast cell disorder are at higher risk for a sudden allergic reaction to the Moderna or Pfizer COVID-19 vaccines. “The public understandably has been concerned about reports of rare, severe… Read More »