Tel Aviv, Israel – In a first, Tel Aviv University researchers have discovered that the production and regulation of mitochondrial organelles in the brain’s nerve cells (neurons) becomes significantly impaired with the deletion of a gene called Gtf2i, one of the 25 genes deleted in Williams syndrome. This impairment is...
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Trieste, Italy – A single treatment with avigbagene parvec, an investigational gene therapy developed by Spur Therapeutics, may help improve bone health in adults living with Gaucher disease, according to new clinical trial data. The findings come from the completed Phase 1/2 GALILEO-1 trial and its ongoing long-term extension study,...
Basel — New results from a one-year Phase III study have confirmed that the investigational biological therapy Ilaris® (canakinumab, formerly ACZ885)[*] produced rapid and sustained remission of symptoms in the majority of children and adults with a rare and potentially life-threatening auto-inflammatory disease called cryopyrin-associated periodic syndrome (CAPS)[1],[2],[3]. The study...
An online symptom management tool that harnesses the problem-solving benefits of expressive writing could help women with ovarian cancer better manage complex symptoms, according to a new study led by a University of Pittsburgh and UPMC nurse-scientist. Published today in the Journal of Clinical Oncology, the study found that patients...
Hattie Foster looks up to the sky. She’s 6 years old. The sun is shining, and she’s buzzing with energy. “Where’s the moon?” she asks whomever is listening. Her older brother, 9-year-old Charlie, climbs across the nearby jungle gym at a Prairie Village playground as her younger brother, 3-year-old Will,...
Primary central nervous system lymphoma (PCNSL) is a rare and aggressive lymphoma with no approved treatment in the U.S.1 In the Phase 2 PROSPECT Study, tirabrutinib demonstrated an overall response rate of 67%, complete response rate of 44%, and a manageable safety profile2 OSAKA, Japan — Ono Pharmaceutical Co., Ltd. (Headquarters:...
OSAKA, Japan — Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President: Toichi Takino; “Ono”) today announced that Ono submitted an application for the manufacturing and marketing approval of ripretinib (DCC-2618), developed by Deciphera Pharmaceuticals, Inc. (“Deciphera”), for the indication of “gastrointestinal stromal tumor that has progressed following cancer chemotherapy.” This...
OSAKA, Japan — Ono Pharmaceutical Co., Ltd. (Headquarters: Osaka, Japan; President and COO: Toichi Takino; “Ono”) announced that efficacy signals for ONO-2808, a S1P5 receptor agonist, were observed in the interim analysis of the Phase 2 clinical trial (ONO-2808-03 study) which assessed the safety and efficacy of ONO-2808 compared to...
PRINCETON, N.J. & ALAMEDA, Calif. — Bristol Myers Squibb and Exelixis, Inc. today announced four-year follow-up results from the CheckMate -9ER trial evaluating Opdivo® (nivolumab) in combination with CABOMETYX® (cabozantinib) vs. sunitinib in patients with previously untreated advanced or metastatic renal cell carcinoma (RCC). Results continued to show superior progression-free...
By Louise Rodino-Klapac – PhD President of Research & Development at Sarepta Therapeutics Cambridge, Mass. – Momentum in Duchenne muscular dystrophy was palpable at the Muscular Dystrophy Association Scientific Congress last month. With nearly 180 abstracts focused on Duchenne alone, spanning pre-clinical research to post-market experience, the field is at...