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…
News
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…
Immune Therapy AO-176 Awarded FDA’s Orphan Drug Designation
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Arch Oncology‘s experimental immune-modulating therapy AO-176 as a potential treatment for…
Riney Foundation’s $40M Gift to Further Research at Dana-Farber
The Paula and Rodger Riney Foundation continues to support the Dana-Farber Cancer Institute in its efforts to advance multiple myeloma research, this time…
Japan OKs Abecma as 1st CAR T-cell for Heavily Treated Myeloma
Japan’s Ministry of Health, Labour, and Welfare has approved Abecma (idecabtagene vicleucel) as its first CAR T-cell therapy for adults with relapsed or refractory…
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