Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
The Orphan Drug Act of 1983 was designed to facilitate the development of orphan drugs, or drugs intended for rare or life-threatening indications. In the United States, rare conditions are…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
When Jen and Will Fox remember their son Dalton, they think about his infectious smile, his passion for adventures, and his absolute adoration of baseball. Dalton, who was diagnosed with…
Salarius Pharmaceuticals has just announced the addition of a new clinical trial site for their study investigating seclidemstat as a treatment for Ewing sarcoma (relapsed or refractory) as well as…
In a press release from late May 2021, biotechnology company CancerVAX, Inc. ("CancerVAX"), which works to develop targeted immunotherapy treatments, shared that it had entered into a sponsored research agreement…
In a recent press release, biopharmaceutical company Salarius Pharmaceuticals, Inc. ("Salarius") shared that it had initiated an expansion stage of a Phase 1/2 clinical trial. Within the trial and the…
Recently, biopharmaceutical company Oncternal Therapeutics ("Oncternal") announced that it has additional clinical data available from its Phase 1 clinical trial. Through the trial, researchers are analyzing an investigational therapy, TK216,…
Cabozantinib has recently been studied as a treatment for Ewing sarcoma and osteosarcoma. It demonstrated antitumor activity in both of these cancers, which was discovered in the second phase of…
According to a story from streetinsider.com, the Joseph Ahmed Foundation and Tyme Technologies, Inc. recently announced that the first patient has been dosed in a phase 2 clinical trial. This…
Oncternal Therapeutics is working on TK216, a drug that is the first in its class to treat Ewing sarcoma. They have recently opened enrollment for Phase 1b of their…
15-year-old George Taylor reached out to NY Giants Running Back Saquon Barkley, telling the star NFL player how he's an inspiration to him for when he undergoes his chemotherapy for Ewing's sarcoma,…
According to a story from BioSpace, the cancer drug company Gibson Oncology, LLC has recently announced that the US Food and Drug Administration (FDA) has awarded Rare Pediatric Disease designation…
9% Copyscape An investigational drug called CLR 131 has received Rare Pediatric Disease Designation from the US FDA for the treatment of Ewing’s sarcoma. The source press release can be…
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