Neurofibromatosis Type
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2025-12-26Clinical Trial Update: Resiliency Training in Adolescents With NF1 and NF2 [NA]
Clinical trial update describing resiliency training for adolescents with neurofibromatosis types 1 and 2; the study is currently active but…
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CLINICAL_TRIALS trial_result · 2025-12-24Clinical Trial Update: Resiliency Training for Adults With Neurofibromatosis Via Live Videoconferencing [NA]
A completed study evaluating resiliency training delivered to adults with neurofibromatosis via live videoconferencing.
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CLINICAL_TRIALS research · 2025-12-18Clinical Trial Update: RASopathy Biorepository
This clinical trial update indicates that the RASopathy Biorepository is currently recruiting participants with various RASopathy-related co…
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CLINICAL_TRIALS research · 2025-12-18Clinical Trial Update: Study of Aspirin in Patients With Vestibular Schwannoma [Phase 2]
Phase 2 clinical trial update for aspirin in vestibular schwannoma patients; the study is currently active but not recruiting.
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CLINICAL_TRIALS research · 2025-12-08Clinical Trial Update: Doxycycline in Cutaneous Schwannoma (NF2) [Phase 1, Phase 2]
A Phase 1/2 clinical trial is evaluating doxycycline for cutaneous schwannomas in Neurofibromatosis Type 2, sponsored by Massachusetts Eye a…
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CLINICAL_TRIALS research · 2025-12-04Clinical Trial Update: PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas [Phase 1]
Phase 1 clinical trial evaluating PAS-004 in adults with Neurofibromatosis Type 1 and related plexiform neurofibromas; currently recruiting.
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CLINICAL_TRIALS trial_result · 2025-12-02Clinical Trial Update: Evaluating Genetic Modifiers of Cutaneous Neurofibromas in Adults With Neurofibromatosis Type 1
A Stanford University–led clinical trial evaluating genetic modifiers of cutaneous neurofibromas in adults with Neurofibromatosis type 1 has…
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CLINICAL_TRIALS trial_result · 2025-11-21Clinical Trial Update: Efficacy of Skin Cooling in Reducing Pain Associated With Non-invasive Treatments of Neurofibromatosis Type 1 Cutaneous Neurofibromas [Phase 1]
Phase 1 trial evaluating whether cooling the skin reduces pain during non-invasive treatments for neurofibromatosis type 1 cutaneous neurofi…
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FDA approval · 2025-11-19FDA Approved: KOSELUGO — Supplemental Application (AstraZeneca Pharmaceuticals LP)
The FDA approved a supplemental application for KOSELUGO (selumetinib) from AstraZeneca Pharmaceuticals LP.
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CLINICAL_TRIALS trial_result · 2025-10-20Clinical Trial Update: Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas [Phase 3]
Phase 3 clinical trial results evaluate the efficacy and safety of selumetinib in adults with neurofibromatosis type 1 and symptomatic, inop…
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CLINICAL_TRIALS research · 2025-10-14Clinical Trial Update: Natural History Study of Cutaneous Neurofibromas in People With NF1
Observational natural history study of cutaneous neurofibromas in people with Neurofibromatosis Type 1; the study is active but not currentl…
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CLINICAL_TRIALS trial_result · 2025-10-07Clinical Trial Update: Efficacy and Safety of REC-2282 in Patients With Progressive Neurofibromatosis Type 2 (NF2) Mutated Meningiomas [Phase 2, Phase 3]
A Phase 2/3 clinical trial evaluating REC-2282 in progressive NF2-mutated meningiomas has been terminated.
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CLINICAL_TRIALS research · 2025-10-03Clinical Trial Update: A Decentralized Clinical Trial to Promote Evidence-Based Care for Underserved Patients With Neurofibromatosis 1 [NA]
A decentralized clinical trial at Massachusetts General Hospital is investigating evidence-based care for underserved patients with Neurofib…
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CLINICAL_TRIALS research · 2025-09-26Clinical Trial Update: Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)
This ClinicalTrials.gov entry describes a National Cancer Institute-sponsored study to surveil malignant transformation of NF1-related perip…
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CLINICAL_TRIALS research · 2025-09-12Clinical Trial Update: Function of the Pigment Epithelium in Patients With Type 1 Neurofibromatosis
A completed clinical trial evaluating the function of the pigment epithelium in patients with Neurofibromatosis Type 1.
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FDA approval · 2025-09-10FDA Approved: KOSELUGO — Original Application (AstraZeneca Pharmaceuticals LP)
FDA approved KOSELUGO (selumetinib) as an original application for pediatric patients with neurofibromatosis type 1 and inoperable plexiform…
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CLINICAL_TRIALS research · 2025-09-09Clinical Trial Update: Identification of Pre-Malignant Lesions In Pediatric Patients With Neurofibromatosis Type 1 Using Novel Magnetic Resonance Imaging Techniques Paired With Artificial Intelligence [NA]
This clinical trial is recruiting pediatric patients with Neurofibromatosis Type 1 to identify pre-malignant lesions using novel MRI techniq…
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CLINICAL_TRIALS research · 2025-09-02Clinical Trial Update: Developing Biomarkers of Plexiform Tumor Burden in Patients With Neurofibromatosis-Type 1
This ongoing clinical trial aims to develop biomarkers to measure plexiform tumor burden in patients with neurofibromatosis type 1.
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CLINICAL_TRIALS trial_result · 2025-08-07Clinical Trial Update: MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas [Phase 2]
Phase 2 clinical trial of the MEK inhibitor mirdametinib for Neurofibromatosis type 1–associated plexiform neurofibromas has posted results.
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CLINICAL_TRIALS research · 2025-07-29Clinical Trial Update: Phase II Study of FCN-159 in NF2 Nerve Sheath Tumors [Phase 2]
Phase II clinical trial of FCN-159 for NF2-related nerve sheath tumors is currently recruiting at the Cancer Institute and Hospital, Chinese…
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CLINICAL_TRIALS trial_result · 2025-07-24Clinical Trial Update: Targeting the Mechanisms Underlying Cutaneous Neurofibroma Formation in NF1: A Clinical Translational Approach.
A completed clinical trial update exploring the mechanisms behind cutaneous neurofibroma formation in neurofibromatosis type 1 using a trans…
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CLINICAL_TRIALS research · 2025-07-03Clinical Trial Update: Early Phase Study Evaluating MEK and MDM2 Inhibition in Patients With NF1 and MPNST [Phase 1, Phase 2]
An early-phase clinical trial is evaluating the combination of a MEK inhibitor and an MDM2 inhibitor in patients with Neurofibromatosis type…
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CLINICAL_TRIALS research · 2025-06-24Clinical Trial Update: Interventions for Reading Disabilities in NF1 [Phase 2]
Phase 2 clinical trial evaluating interventions for reading disabilities in Neurofibromatosis Type 1 is currently active but not recruiting.
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CLINICAL_TRIALS research · 2025-06-17Clinical Trial Update: Follow-up Study to Evaluate the Safety and Efficacy of FCN-159 in Pediatric Participants With Neurofibromatosis Type 1 [Phase 1, Phase 2]
A registry update describing a Phase 1/Phase 2 follow-up study evaluating FCN-159 in pediatric patients with Neurofibromatosis Type 1, focus…
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CLINICAL_TRIALS research · 2025-06-10Clinical Trial Update: Non-interventional Study of Patients With PN NF1 Starting Selumetinib in Russia
An observational non-interventional study in Russia evaluating outcomes for patients with plexiform neurofibromatosis type 1 starting selume…