List of Rare Diseases Tops 10,000 – and Is Growing – New Analysis Finds
Note: This story was updated July 13, 2022, to correct the name of Rare-X’s CEO Charlene Son Rigby. Nonprofits, scientists, governmental organizations, and…
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Talquetamab Named a Breakthrough Therapy for Hard-to-treat Myeloma
Talquetamab, a first-in-class antibody treatment being developed by Janssen Research & Development, has been granted breakthrough therapy designation by the U.S. Food and Drug Administration (FDA)…
1st CAR T-Cell Therapy in China for Myeloma Now Under Review
The China National Medical Products Administration (NMPA) has agreed to review an application for Equecabtagene autoleucel — the country’s first chimeric antigen receptor (CAR) T-cell…
Novel Immunotherapy QXL138AM Wins Orphan Drug Status in US
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Nammi Therapeutics’ lead immunotherapy candidate QXL138AM for multiple myeloma. The…
CYT-338 Shows Anticancer Activity in Preclinical Tests
CYT-338, an investigational myeloma therapy designed to simultaneously bind to cancer cells and activate immune cells called natural killer cells, has shown promising anti-cancer…
CAR T-cell Therapy Safe, Effective in Treatment-resistant Myeloma: Study
A CAR T-cell therapy called CART-ddBCMA that targets the protein BCMA — produced at high levels in myeloma cells — safely led to lasting…
Ninlaro as Oral Combo Therapy Fails at Trial in Heavily Treated Patients
Adding Ninlaro (ixazomib) to cyclophosphamide and dexamethasone does not improve progression-free survival for multiple myeloma patients who relapsed after several lines of treatment,…
Agreement Reached to Develop MMP-13 Inhibitors to Treat Myeloma
SteroTherapeutics has entered into an exclusive license agreement with Moffitt Cancer Center and Florida Atlantic University Research Corporation to develop matrix metalloproteinase 13…
Organizations Rally to Help Ukrainian Rare Disease Patients
A Russian military plane crash near Tetiana Zamorska’s home in Kyiv, Ukraine, was a sign that it was time for her and her family to leave.
Europe Awaits Proposed New Framework for Sharing Health Data
The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the…
