#RAREis Representation Program Promotes Equity, Diversity
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…
News
FDA Places HPN217, Immunotherapy for Advanced Disease, on Fast Track
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 …
Rare Disease Day Panel Opens Window to Patient Experience
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…
FDA Approves Cilta-cel as Carvykti for Heavily Treated Myeloma
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…
Surge in Telehealth Brings Access, Convenience to Rare Disease Patients
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…
IMF Opening ‘Action’ Campaign to Mark Myeloma Awareness Month
The International Myeloma Foundation (IMF) is opening its annual Myeloma Action Month (MAM) campaign with a variety of events and undertakings…
Q&A With RARE-X Disease Data Platform Founder, Nicole Boice
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…
More Studies Confirm Hefty Economic Burden of Rare Diseases
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.
FDA Designates CT103A, CAR T-cell Therapy, an Orphan Drug
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…
Xpovio-Kyprolis Combo Shows Good Response in Hard-to-treat Myeloma
Xpovio (selinexor) combined with Kyprolis (carfilzomib) and low-dose dexamethasone leads to strong and durable responses in heavily pretreated multiple myeloma patients, results…
