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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SAN DIEGO, Calif. — Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced the first presentation of new two-year data from the Phase 3 CAHtalyst® Adult study demonstrating sustained, substantial reductions in glucocorticoid (GC) doses in adults with classic congenital adrenal hyperplasia treated with CRENESSITY® (crinecerfont), with approximately 70% of patients achieving GC doses within the physiologic range. These...
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) today announced that the first patient has been dosed in the BALANCE-CAH Phase 2/3 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) in children and...
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 European Commission (EC) has approved PALSONIFY® (paltusotine), the first once-daily, oral, selectively-targeted somatostatin receptor type 2 nonpeptide agonist, for the treatment of adult patients with acromegaly. “The European Commission’s decision to approve Palsonify reflects the strength of the clinical data and...