Magnetic resonance imaging (MRI) has been used to study neuromyelitis optica spectrum disorders (NMOSD) in the past. Specifically, it is used to visualize the involvement of the spinal cord's central…
In the past, research has suggested that there was a genetic component to essential tremor (ET), a nervous system disorder. According to Medscape, researchers recently expanded on this understanding by…
Karyopharm Therapeutics has just announced that Eltanexor, their investigative therapy for myelodysplastic syndromes (MDS), has been given Orphan Drug Designation by the FDA. This designation means that the FDA believes…
Jordan's Guardian Angels, a nonprofit organization that supports the Jordan's syndrome community, has recently announced a new scholarship that will honor children that have died as a result of being…
It is common practice to treat multiple myeloma patients with a stem cell transplant. In the GENESIS trial, the stem cells were autologous (from the patient’s own peripheral blood or bone…
A Phase 3 clinical trial for advanced biliary tract cancer (BTC) has found that there is an improved overall survival rate when patients are given the immunotherapy durvalumab in combination…
In a late January 2022 news release, gene therapy company Taysha Gene Therapies, Inc. ("Taysha") shared that trial data was now available on TSHA-120. The therapy, designed to treat giant…
On January 27, 2022, The EveryLife Foundation for Rare Diseases, in partnership with the Biotechnology Innovation Organization (BIO), National Health Council, and Pharmaceutical Research and Manufacturers of America (PhRMA), hosted…
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