Brelovitug has received breakthrough therapy designation from the US Food and Drug Administration.
Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis ...
Muscular Dystrophy Association Marks Rare Disease Day by Spotlighting Community Stories Driving Progress in Research, Care, ...
Ashleigh McGeogh's daughter Delilah was diagnosed with Kawasaki disease, a condition that is the leading cause of acquired ...
XCells Biotechnologies ("iXCells"), a leading provider of human cell-based solutions and custom iPSC services, and Rosebud ...
Papzimeos, an HPV-specific immunotherapy, is the first FDA-approved treatment for recurrent respiratory papillomatosis, ...
By determining the structure of the deposits responsible for transthyretin amyloidosis through a simple skin biopsy, scientists at UNIGE are paving the way for a new diagnostic method for ...
A drug being developed by Boehringer Ingelheim for COVID-19 and reinvented as a potential therapy for kidney disease – and has now hit the mark in a phase 2 trial. TRPC6 inhibitor apecotrep (BI 764198 ...
UCL scientists have grown mini-stomachs in the lab that closely mimic human organs, providing a powerful new way to ...
Dr Sudheendra Rao N R from the Organization for Rare Diseases India reveals that approximately 70 million are affected by ...
A 2-year-old boy died from a rare disease with flu-like symptoms, just weeks before his third birthday. Hudson Hughie Martin, ...
Some results have been hidden because they may be inaccessible to you
Show inaccessible results