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 discussions on Rare Disease Day 2022, which brings international awareness about the more than 300 million people living with rare disorders. Part of that…
The International Myeloma Foundation (IMF) is opening its annual Myeloma Action Month (MAM) campaign with a variety of events and undertakings to raise disease awareness across the community and the public-at-large. “Action” is theme of this year’s monthlong event, held each March. “The focus for this year’s…
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 disorders, discover new ones, and work toward finding treatments. It was spun out of the work that Nicole Boice, founder and chief engagement officer of…
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 Innovent Biologics are developing to treat relapsed or refractory multiple myeloma. Orphan drug designation is given to therapies with a potential to substantially improve…
Xpovio (selinexor) combined with Kyprolis (carfilzomib) and low-dose dexamethasone leads to strong and durable responses in heavily pretreated multiple myeloma patients, results from a Phase 1/2b trial show. The study, “Once weekly selinexor, carfilzomib and dexamethasone in carfilzomib non-refractory multiple myeloma patients,” was published…
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 relapsed or refractory multiple myeloma. This designation, given to therapies with the potential to improve care for conditions that affect fewer than 200,000 people…
The Paula and Rodger Riney Foundation continues to support the Dana-Farber Cancer Institute in its efforts to advance multiple myeloma research, this time with a $40 million grant. This gift — the largest single award to myeloma research in Dana-Farber’s history — brings the foundation’s donations to the institute…
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 multiple myeloma who received at least three prior therapies. This includes patients treated at least with one immunomodulatory drug (IMiD), one proteasome inhibitor…
A model that may better classify multiple myeloma (MM) using genetic sequence data from hundreds of patients has been developed. The model can identify specific genes and genetic alterations responsible for disease subtypes related to a risk of relapse, and it may contribute to the identification of targeted therapies,…
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