A small subset of vulnerable DNA sites can make cancer cells highly sensitive to targeted drugs called PARP inhibitors, a new study shows. Led by researchers at NYU Langone Health and its Perlmutter Cancer Center, the work revolved around failures…
Presented by The Community Oncology Alliance In part 2, Susan M. Wescott, RPh, MBA, discusses how payers navigate evidence gaps, prior authorization, and access to rare disease therapies. Because rare disease therapies often launch without all necessary data, payers must rely on…
CHAMPAIGN, Ill. — A particular signaling protein in the brain could be a therapeutic target for neurological disorders arising from the deletion of the autism-linked gene MEF2C, University of Illinois Urbana-Champaign researchers found in a new mouse study. Deletions or mutations to MEF2C are…
Research By: Maurizio Macaluso, MD, DPH | Michael Wagner, PhD | William R. Otto, MD Post Date: November 4, 2026 | Publish Date: Rare diseases may affect relatively few people individually, but together they impact millions of children and adults. Finding effective treatments can be especially…
Furthermore, the team discovered that an X-linked transcriptional activator called ZFX, which escapes X-chromosome inactivation and is expressed at higher levels in females, acts as a master regulator of this convergent network—uncovering the molecular machinery underlying the female protective…
OncLive The panelists recommend germline and somatic testing for every patient with metastatic prostate cancer and germline testing for more aggressive localized disease, where results primarily inform cascade testing of relatives rather than treatment They acknowledge ongoing disagreement over testing…
CHAMPAIGN, Ill. — A particular signaling protein in the brain could be a therapeutic target for neurological disorders arising from the deletion of the autism-linked gene MEF2C, University of Illinois Urbana-Champaign researchers found in a new mouse study. Deletions or mutations to MEF2C are…
Molecular Psychiatry (2026) Cite this article Given that multiple proteins coordinate functionally in a cell, there are logical bottlenecks where distinct clusters interact utilizing minimal proteins. These connector proteins were analyzed based on participation coefficient, impact score, and degree: The impact…
Scientists at Boston Children’s Hospital have found that the ZFX gene regulates the activity of many autism-related genes and may be one of the factors behind women’s relative protection from severe symptoms of this condition. The results of the study,…
Genetic interaction networks, such as the one pictured here, allowed researchers to predict functions for previously uncharacterized genes. Newswise — Rockefeller scientists have created the most comprehensive map to date of how essential genes interact in Mycobacterium tuberculosis (Mtb), the bacterium…
Orphan conditions, registries, gene testing, and family support networks.
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