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central nervous system disorder - Clinical Profile, Genes & Research | Kisho | Kisho
Rare Disease
central nervous system disorder
MONDO:0002602
Also known as:CNS disordercentral nervous diseasecentral nervous system disease or disorder
Patient Orgs1 organizations · The Wolverine Foundation
Diagnosis
Biomarker and diagnostic research for central nervous system disorder has been reported in the published literature.
Research Landscape
Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.
33 clinical trials registered, 13 recruiting. Interventions under study include drug therapy, other interventions, and medical devices. Pipeline includes 1 PHASE3, 2 PHASE2, 2 PHASE1. Research is primarily sponsored by academic and government institutions.
Diseases are connected by shared genes, phenotypes, therapeutic targets, and MONDO disease hierarchy. The Rare badge reflects Kisho's rarity classification — scored from authoritative sources including OMIM, Orphanet, orphan drug designations, clinical trial activity, and published prevalence data.
→Are any of the 33 active clinical trials a good fit for my situation?
→Are there specialists or centers of excellence for this condition in my area?
→How can I connect with The Wolverine Foundation and other patient organizations for support?
FAQ
Frequently Asked Questions
Common questions about central nervous system disorder
Central nervous system disorder is a condition that affects the brain and spinal cord, with the underlying cause linked to changes in the MED17 gene. Although precise prevalence estimates are not available, this condition is recognized as involving the central nervous system. The disorder may present at various ages, and its clinical course can vary considerably between individuals. Ongoing research continues to clarify how MED17 contributes to the condition, and care strategies are evolving as our understanding improves.
Specific clinical features for central nervous system disorder have not been well defined in the available data. Patients may experience a range of neurological symptoms, and the presentation can differ markedly from one person to another. Not all individuals experience all features, and severity varies considerably. Because detailed characteristic findings are not provided, evaluation and monitoring by clinical specialists remain important.
The central nervous system disorder is associated with genetic alterations in the MED17 gene, which plays an important role as part of the mediator complex in regulating gene expression. The precise genetic mechanisms and inheritance pattern have not been fully detailed, and further investigation is needed to understand how variants in MED17 lead to the observed symptoms. Families are encouraged to seek genetic counseling to discuss the implications of genetic findings and potential risks for future pregnancies. Associated genes include MED17.
Study of Silevertinib With Temozolomide for the Treatment of Newly Diagnosed GBM With Unmethylated MGMT and EGFRvIII
PHASE2
Black Diamond Therapeutics, Inc.
RECRUITING
254 publications have been identified in PubMed for central nervous system disorder. Kisho has analyzed 117 by research type. Research spans Review / Meta-Analysis (28%), Basic Science / Preclinical (28%), and Case Report / Case Series (15%).
So did investment firms, smaller biotechs, law firms, and banks. With Big Pharma firms readying to buy new assets in the face of looming patent cliffs, all kinds of partnerships are fair game: licensing deals, co-development agreements, and outright acquisitions. It’s not unusual for the firm’s partnerships to span a decade or more. “I think we really want to work with them and bring something forward, rather than buy a baked cake later,” he said. Boehringer on Tuesday announced a new collaboration with the antibody firm ComboTope Therapeutics to develop cancer drugs. Sanofi, the French pharmaceutical giant, is looking for deals in the immunology, rare disease, and vaccine spaces, according to Brian Bronk, head of business development, who spoke on a panel about dealmaking on Tuesday. The law subjects outgoing US investments to reviews by the US Departments of Treasury and Defense. If passed, the updated legislation would likely dampen the spate of US-China biotech deals, which recently include a $15.2 billion co-development agreement between Bristol Myers Squibb and Hengrui and a $10.5 billion partnership between Pfizer and Innovent. Maha Radhakrishnan, executive partner for private equity at Sofinnova Partners, told C&EN that she is interested in drug candidates for cancer, immunology and inflammation, cardiometabolic and renal diseases, central nervous system disorders, and rare diseases. San Diego—Pharmaceutical executives and biotech investors spent their time on the prowl at the BIO International Convention this week, searching for promising new drug candidates as money starts moving through the sector again.
METiS TechBio secures $269M via Hong Kong listing with $148M cornerstone commitments. Biotech funding milestone revealed. METiS TechBio, headquartered in Hong Kong, is an AI-driven drug delivery company with a pipeline spanning oncology, immunology, central nervous system disorders, and metabolic diseases. The company reported revenue of RMB 105 million in 2025, primarily from an upfront payment related to its MTS-004 product partnership, and cumulative R\&D expenditure of approximately RMB 270 million from 2023 to 2025. METiS TechBio (HKEX: 7666) listed on the Hong Kong Stock Exchange on May 13, 2026, raising over HKD 2.1 billion (USD 269 million) in what the company described as the largest healthcare IPO fundraising on HKEX so far in 2026. In addition to BlackRock, cornerstone investors included UBS Asset Management Singapore, Mirae Asset, ORIX Corporation, Guofengtou Innovation Investment Fund, Deerfield, RTW, Lake Bleu Capital, Walden International, Hillhouse Capital, IDG Capital, GF Fund, ICBC UBS Asset Management, China Asset Management, and Fullgoal Fund. Pre-IPO private financing rounds were not disclosed.
A recent study published in PubMed explores the dissemination patterns of central nervous system Burkitt lymphoma, specifically from the fornix to the ventricles. This research may provide insights into the disease's progression and potential treatment strategies.
Ligand’s acquisition of XOMA Royalty expands the royalty model, supporting late-stage drug development and diversifying access to oncology and rare disease therapies. Expanded royalty platforms can recycle capital into emerging pipelines, potentially accelerating development timelines for smaller biotechs while shifting value capture toward diversified royalty holders.SHOW MORE · Ligand’s acquisition of XOMA Royalty expands the royalty model, supporting late-stage drug development and diversifying access to oncology and rare disease therapies. Portfolio breadth expands across oncology, ophthalmology, CNS, and rare diseases, aiming to reduce single-asset volatility through stage and modality diversification. The expanded portfolio spans multiple therapeutic areas, including oncology, ophthalmology, central nervous system disorders, and rare diseases. https://www.wilmerhale.com/en/insights/client-alerts/20240904-royalty-financings-and-similar-revenue-monetizations-surge-in-difficult-life-sciences-fundraising-environment · Fleming C, Levin G, Rahmani F. Alternative sources of capital for biotechs.
