This can be more challenging with the smaller patient populations in rare diseases. But the struggle doesn’t end when the trial does. Instead, the focus is on what comes next: the drug has just ...

Diagnosed with Ewing sarcoma at 9, Lucy Spada beat the odds and survived. Her family started the Little Warrior Foundation to ...

The PSA, airing nationally, represents how MDA galvanizes a global community united in the fight against rare diseases. Narrated with a dynamic script, it highlights MDA’s role in advancing ...

PHILADELPHIA, Feb. 6, 2025 /PRNewswire/ -- This year marks a monumental milestone for NephCure: 25 years of driving hope, innovation, and progress in the fight against rare kidney disease (RKD).

Historically, the fight against rare diseases has been hindered by diagnostic delays, limited access to data, and limited treatment options. But AI is changing that. With increasing FDA support ...

This brave little girl has been dealt a very tough start to life after being diagnosed with an extremely rare lung disease.

Scientists have uncovered a mechanism for reinvigorating the immune system to stop it from flagging when it’s fighting ...

"In Bulgaria, there is discrimination both on the basis of disease (rare or frequent diagnosis) and age." Bulgaria's Commission for Protection against Discrimination has announced that patients ...

[101] In light of this, it is becoming critical to improve our understanding of aging and its associated diseases to support existing healthcare systems coping with the burden of diseases and to ...