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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’...
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, 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...
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...
Basel, Switzerland – New research presented early ahead of this year’s European Congress of Clinical Microbiology and Infectious Diseases (ECCMID 2024, Barcelona, 27-30 April) from a team of researchers in the Netherlands shows how the latest CRISPR-Cas gene editing technology can be used to eliminate all traces of the HIV...
SAN FRANCISCO, CA  —  The gene-editing technology CRISPR shows early promise as a therapeutic strategy for the aggressive and difficult-to-treat brain cancer known as primary glioblastoma, according to findings of a new study from Gladstone Institutes. Using a novel technique they’ve dubbed “cancer shredding,” the researchers programmed CRISPR to zero-in...
SAN DIEGO – Cardea Bio, a Tech+Bio company integrating molecular biology with semiconductors electronics via graphene-based biology-gated Cardean Transistors, announced today that its Chief Science Officer, Dr. Kiana Aran, and collaborators published a paper entitled “Discrimination of single-point mutations in unamplified genomic DNA via Cas9 immobilized on a graphene field-effect...
TUCSON, Ariz. — Critical Path Institute (C-Path) is excited to announce the launch of the Critical Path for Alpha-1 Antitrypsin Deficiency (CPA-1) Consortium. The CPA-1 consortium aims to accelerate drug development for alpha-1 antitrypsin deficiency (AATD), a rare disease that affects individuals and families worldwide. This will be achieved by...