Inspired by the rare disease community’s passionate drive to advance policies that provide equitable access to care, UCB ...

Through policy framework shifts, appeals to the court for rare disease funds, and starting a centre in Bengaluru, Prasanna ...

Alkaptonuria is a rare genetic disorder that causes black urine due to homogentisic acid buildup, leading to joint pain, ...

Addison’s disease is a rare autoimmune disorder where the adrenal glands don’t produce enough vital hormones, causing fatigue ...

Christian Fellowship Center Lead Pastor Brian Dube is making a miraculous recovery from a case of near-fatal paralysis from a ...

Cristina Brennan can hear the clock ticking. At the end of the year, her 12-year-old son Tristan will no longer have free ...

In honor of Rare Disease Day on February 28, 2025, we will publish a series of posts throughout the month on As Prescribed ...

Rare Disease Day is February 28. Primary sclerosing cholangitis or PSC is a rare liver disease characterized by progressive ...

Advocacy groups urge Health Ministry to intervene urgently in delayed funding for rare disease treatments, impacting patients ...

After combing through 4,000 existing medications, an artificial intelligence tool helped uncover one that saved the life of a patient with idiopathic multicentric Castleman's disease (iMCD).

An England-wide study of over 58 million people has identified eight rare diseases that carry significantly increased risks for COVID-19-related mortality in fully vaccinated individuals.

In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches -- gene therapy ...