Researchers from Osaka University develop a collaborative process for involving patients, families and policymakers in the conversation around priorities for rare-disease research Osaka, Japan – Patients with rare diseases have traditionally been the subjects of medical research. However, in recent years, their role has begun to shift from ‘research participants’...
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Baltimore, Maryland – The incidence of Creutzfeldt-Jakob disease (CJD), a universally fatal prion disease that progresses rapidly, rose consistently from 2007 to 2020, a study of death certificates showed. CJD codes on death certificates totaled 5,882 during that period, with 51.2% of cases occurring among women, according to John Probasco,...
SAN DIEGO, Calif. — Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced that the U.S. Food and Drug Administration (FDA) approved PALSONIFYTM (paltusotine) for the first-line treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom surgery is not an option. PALSONIFY, a selectively-targeted somatostatin receptor type 2...
SAN DIEGO, Calif. — Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced that the first patient has been dosed in the CALM-CAH Phase 3 trial evaluating investigational candidate atumelnant, a novel, once-daily, oral adrenocorticotropic hormone (ACTH) receptor antagonist candidate for the proposed treatment of classic congenital adrenal hyperplasia (CAH). “Dosing the first...
SAN DIEGO, Calif. — Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced the first patient has been randomized in the pivotal Phase 3 CAREFNDR trial, a multicenter, randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of once-daily, oral paltusotine in adults with carcinoid syndrome due to well-differentiated neuroendocrine tumors. The Phase...
SAN DIEGO, CA — Crinetics Pharmaceuticals, Inc. today announced positive initial findings from its ongoing open-label Phase 2 carcinoid syndrome (CS) study of paltusotine, an oral, once-daily investigational compound being developed for the treatment of acromegaly and CS. “We are very encouraged by these strong initial findings in our Phase...
SAN DIEGO, Calif. — Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) a clinical stage pharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for endocrine diseases and endocrine-related tumors, today announced positive topline results from PATHFNDR-2, the second of two Phase 3 studies evaluating the efficacy and safety of...
SAN DIEGO, Calif. — Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) for atumelnant, a novel, once-daily oral adrenocorticotropic hormone (ACTH) receptor antagonist candidate for the proposed treatment of classic congenital adrenal hyperplasia (CAH). Atumelnant is the first and...
Brave teenager Vicki Wood is felled by terrifying seizures whenever she feels sad, angry, scared – or HAPPY. Her legs buckle and she crashes to the ground up to a dozen times a day. And the 18-year-old now tries to be as boring as possible in a desperate bid to...
Tel Aviv, Israel – Researchers from Tel Aviv University utilized CRISPR to cut a single gene from cancer cells of head and neck tumors – and successfully eliminated 50% of the tumors in model animals. This groundbreaking study was led by Dr. Razan Masarwy, MD, Ph.D. from the lab of Prof....