New trials, publications, preprints and grants on this target, month by month.
Trial
Combination of Avutometinib and Defactinib for Treatment of Relapsed, Refractory, Metastatic, or Unresectable Malignant Peripheral Nerve Sheath Tumor
1 Jan 2027· PHASE2 · NOT_YET_RECRUITING· CTGOV
Trial
Efficacy of Mirdametinib Alone or Combination With Radiotherapy for Germline and Sporadic NF1-Altered High-Grade Glioma
1 Jan 2027· PHASE2 · NOT_YET_RECRUITING· CTGOV
Publication
17q11.2 Duplication (includes NF1 gene): A Scoping Review.
5 Nov 2026· Genetics in medicine : official journal of the American College of Medical Genetics· PUBMED
Publication
Identification of CD74-Positive Antigen-Presenting Glioma Cells in Primary Human Tumors and Murine Models of NF1 High-Grade Glioma.
2 Oct 2026· Molecular cancer therapeutics· PUBMED
Publication
Exceptional and Durable Response to Selpercatinib in a Patient with Apparently RET-Negative, HRAS-, and NF1-Mutated Metastatic Medullary Thyroid Carcinoma.
1 Oct 2026· Thyroid : official journal of the American Thyroid Association· PUBMED
Trial
Cognitive Intervention for Children and Adolescents With NF1
28 Sept 2026· NA · NOT_YET_RECRUITING· CTGOV
Publication
Neurofibromatosis 1 (NF1) gene testing reveals rising variant allele fraction as an early warning sign of juvenile myelomonocytic leukemia.
11 Sept 2026· European journal of medical genetics· PUBMED
Trial
A study of Mirdametinib in Infants and Toddlers under 24months of age with Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PNs)
3 Sept 2026· PHASE1 · RECRUITING· CTIS
Publication
Single-Cell Analysis Guides Organoid CRISPR Screening to Reveal NF1 as a Gatekeeper of Cervical Squamous Malignant Transformation.
3 Sept 2026· Cancer research· PUBMED
Publication
Colon cancer in a patient with a mosaic monoallelic germline pathogenic NF1 gene variant.
3 Sept 2026· Exploration of neuroscience· PUBMED
Publication
NF1 alterations as potential biomarker in acral melanoma: looking beyond low tumor mutational burden.
25 Aug 2026· Melanoma research· PUBMED
Trial
Trial of Selumetinib and Bromodomain Inhibitor With Durvalumab for Sarcomas
4 Aug 2026· PHASE2 · RECRUITING· CTGOV
Trial
Gait in Young Children With NF1
1 Aug 2026· NA · NOT_YET_RECRUITING· CTGOV
Publication
NF1 Leucine Rich Domain-Derived Extracellular Vesicles Remodel the Glioblastoma Immune Microenvironment via an ADAM17-Associated Inflammatory Program.
1 Aug 2026· Journal of extracellular vesicles· PUBMED
Publication
Spatial proteomic profiling reveals an association between NF1-Ras/Raf/MEK signaling and immune cell infiltration in locally advanced triple-negative breast cancer.
28 Jul 2026· Translational oncology· PUBMED
Publication
Sleep Health and Quality of Life in Children and Adolescents with NF1: A Biopsychosocial Perspective.
22 Jul 2026· Cancers· PUBMED
Publication
Case Report: Schwann cell reprogramming and PDGF-driven nerve hypertrophy in an NF1 patient with CIDP-like autoimmunity.
17 Jul 2026· Frontiers in immunology· PUBMED
Publication
Epilepsy, Cognitive, and Behavioral Outcomes in Neurocutaneous Syndromes: A Comparative Review of NF1, TSC, and Sturge-Weber Syndrome.
9 Jul 2026· Children (Basel, Switzerland)· PUBMED
Publication
Cognitive function in adult NF1 patients without other cognitive disorders: a cross-sectional study.
3 Jul 2026· Orphanet journal of rare diseases· PUBMED
Publication
Durable responses to long-term selumetinib in Chinese pediatric NF1 patients with inoperable plexiform neurofibromas.
18 Jun 2026· Frontiers in pharmacology· PUBMED
Publication
NF1 mutation may be associated with lung-tropic metastasis in cutaneous melanoma: a genomic analysis of 520 patients.
6 Jun 2026· Clinical & experimental metastasis· PUBMED
Trial
Early Phase Study Evaluating MEK and MDM2 Inhibition in Patients With NF1 and MPNST
1 Jun 2026· PHASE1_PHASE2 · RECRUITING· CTGOV
Publication
NF1 loss in estrogen receptor-positive breast cancer induces osteoclast formation and immunosuppression to promote bone metastasis.
29 May 2026· Cancer letters· PUBMED
Publication
Exceptional response to everolimus in an NF1-deficient recurrent endometrioid adenocarcinoma: a case report.
25 May 2026· Frontiers in oncology· PUBMED
Publication
Prognostic impact of NF1 mutation in Korean cohort with glioblastoma.
21 May 2026· Frontiers in neurology· PUBMED
Publication
Integrative Genomic and Functional Analysis Reveals NF1 Loss as a Modifier of DNA Damage and Replication Stress Responses in Ovarian Cancer.
18 May 2026· Human mutation· PUBMED
Publication
Melanoma with Blue Nevus-like Morphology Harboring NRAS and NF1 Mutation.
17 May 2026· Journal der Deutschen Dermatologischen Gesellschaft = Journal of the German Society of Dermatology : JDDG· PUBMED
Publication
Bridging the Diagnostic Gap in Neurofibromatosis Type 1: When to Consider NF1 cDNA Sequencing.
13 May 2026· Pediatric dermatology· PUBMED
Publication
Is cognitive profiling in NF1 still optional? A systematic review of current assessment practices.
30 Apr 2026· Frontiers in neurology· PUBMED
Publication
Regulation and Implementation of Apoptosis in Melanoma Tumor Cells with BRAF, NRAS, and NF1 Gene Mutations.
22 Apr 2026· Current issues in molecular biology· PUBMED