The therapy Sarclisa (isatuximab) extended survival by nearly seven months in relapsed/refractory multiple myeloma patients also given Pomalyst (pomalidomide) and dexamethasone as standard…
Horizon Therapeutics has launched its #RAREis Representation program aimed at increasing diversity, equity, and inclusion among patients with rare diseases. There are about 400 million…
The U.S. Food and Drug Administration (FDA) has given fast track designation to Harpoon Therapeutics’ HPN217 as a potential immunotherapy for people with relapsed, refractory …
Bionews, the publisher of this website, hosted a virtual panel discussion on Rare Disease Day 2022, taking a deeper dive into what it’s like…
The U.S. Food and Drug Administration (FDA) has approved the CAR T-cell therapy Carvykti (ciltacabtagene autoleucel) — formerly known as cilta-cel — for certain…
A rare disease puts an economic burden on the patients, families, and caregivers that it affects, and will no doubt be an integral part of…
The International Myeloma Foundation (IMF) is opening its annual Myeloma Action Month (MAM) campaign with a variety of events and undertakings…
The nonprofit RARE-X is creating an easily-accessible, centralized data hub for all rare disease patient data that can help researchers answer questions about existing…
It’s been nearly a year since the EveryLife Foundation for Rare Diseases released its expansive report finding the total economic burden of rare disorders in the U.S. to be nearly $1 trillion.
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to CT103A, an experimental CAR T-cell therapy that IASO Biotherapeutics and…