Neurofibromatosis Type
📧 Sign up to get email alerts when something new happens for Neurofibromatosis Type.
Sign up freeTop drugs studied for this disease
-
EMA approval · 2026-08-13EMA Authorised: Ezmekly (mirdametinib) — Merck Europe B.V.
EU authority authorised Ezmekly (mirdametinib) for Neurofibromatosis type 1 with neurofibromas, including plexiform neurofibroma, under cond…
-
EMA approval · 2026-07-01EMA Authorised: Koselugo (selumetinib sulfate) — AstraZeneca AB
Koselugo (selumetinib sulfate) has been authorised by the EMA for Neurofibromatosis Type 1 under conditional approval as an orphan medicine.
-
CLINICAL_TRIALS trial_result · 2026-06-05Clinical Trial Results: Antioxidant Therapy With N-acetylcysteine for Children With Neurofibromatosis Type 1 [Phase 2]
Phase 2 trial assessing N-acetylcysteine as antioxidant therapy in children with Neurofibromatosis Type 1 has completed with results posted.
-
CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors [Phase 1, Phase 2]
The National Cancer Institute reports a Phase 1/Phase 2 trial of selumetinib (AZD6244 hydrogen sulfate) for children with neurofibromatosis …
-
CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neurofibromas [Phase 1, Phase 2]
The National Cancer Institute is sponsoring Phase 1/2 trials evaluating the CDK4/6 inhibitor abemaciclib for Neurofibromatosis type I–relate…
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: Natural History Study of Patients With Neurofibromatosis Type 2
Natural history study of patients with Neurofibromatosis Type 2, currently recruiting to observe disease progression and clinical features o…
-
CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)
This clinical trial update describes the development and validation of patient-reported outcome measures for Neurofibromatosis type 1 and pl…
-
CLINICAL_TRIALS trial_result · 2026-04-24Clinical Trial Update: Transformation of Plexiform Neurofibromas to Malignant Peripheral Nerve Sheath Tumors in Neurofibromatosis Type 1 [Phase 1]
A Phase 1 trial investigating the transformation of plexiform neurofibromas into malignant peripheral nerve sheath tumors in Neurofibromatos…
-
CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor ... [Phase 1]
Phase I trial of pexidartinib (TURALIO) in children and young adults with refractory leukemias and solid tumors including NF1-associated ple…
-
CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: Study of Disease Severity in Adults With Neurofibromatosis Type 1 (NF1)
This clinical-trial update reports the completion of a study evaluating disease severity in adults with Neurofibromatosis Type 1 and Legius …
-
CLINICAL_TRIALS research · 2026-04-23Clinical Trial Update: Familial Investigations of Childhood Cancer Predisposition
An ongoing St. Jude-led clinical trial is investigating familial cancer predisposition syndromes in children and is currently recruiting.
-
CLINICAL_TRIALS research · 2026-04-22Clinical Trial Update: Assessing the Efficacy of Repeat, Monthly Treatments of Deoxycholate for NF1 Associated Cutaneous Neurofibromas (cNFs) [Phase 1]
A Phase 1 study at Massachusetts General Hospital is evaluating repeat monthly injections of sodium deoxycholate for cutaneous neurofibromas…
-
EMA approval · 2026-04-21EMA Authorised: Ezmekly (mirdametinib) — SpringWorks Therapeutics Ireland Limited
The European Medicines Agency authorised Ezmekly (mirdametinib) for neurofibromatosis type 1 with plexiform neurofibromas, under conditional…
-
CLINICAL_TRIALS research · 2026-04-16Clinical Trial Update: Prevalence, Clinical Characteristics, Progression, and Management of Neurofibromatosis Type 1 in Egypt (NF1-Egy)
AstraZeneca-sponsored study in Egypt is currently recruiting to investigate the prevalence, clinical characteristics, progression, and manag…
-
CLINICAL_TRIALS research · 2026-04-15Clinical Trial Update: A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia [Phase 1, Phase 2]
A Phase I/II clinical trial evaluating the combination of trametinib and azacitidine for newly diagnosed juvenile myelomonocytic leukemia (J…
-
CLINICAL_TRIALS trial_result · 2026-04-14Clinical Trial Update: Natural History and Biology of Skin Neurofibromas in Neurofibromatosis Type 1
This is an update on a completed observational clinical trial studying the natural history and biology of skin neurofibromas in Neurofibroma…
-
CLINICAL_TRIALS research · 2026-04-13Clinical Trial Update: Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma [Phase 1, Phase 2]
Selumetinib is being studied in pediatric patients with recurrent or refractory low-grade glioma across Phase 1 and Phase 2 trials; the stud…
-
CLINICAL_TRIALS research · 2026-04-13Clinical Trial Update: Efficacy of Mirdametinib Alone or Combination With Radiotherapy for Germline and Sporadic NF1-Altered High-Grade Glioma [Phase 2]
Phase 2 trial evaluating Mirdametinib alone or with radiotherapy for germline and sporadic NF1-altered high-grade glioma; status is not yet …
-
CLINICAL_TRIALS research · 2026-04-13Clinical Trial Update: Mirdametinib Monotherapy in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF). [Phase 1, Phase 2]
Clinical trial update indicating recruitment for Mirdametinib monotherapy in adults with Neurofibromatosis type 1 and cutaneous neurofibroma…
-
CLINICAL_TRIALS research · 2026-04-13Clinical Trial Update: A Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma [Phase 3]
Phase 3 trial comparing selumetinib to carboplatin/vincristine in patients with neurofibromatosis type 1-associated low-grade glioma, with t…
-
CLINICAL_TRIALS research · 2026-04-09Clinical Trial Update: A Study to Evaluate the Feasibility of a Physiologic Biomarker to Assess Pain and Other Sensory Problems Using Pupillometry in Participants With Neurofibromatosis Type 1 (NF1) [NA]
An ongoing study to evaluate the feasibility of using pupillometry as a physiologic biomarker to assess pain and other sensory problems in p…
-
CLINICAL_TRIALS research · 2026-04-09Clinical Trial Update: Development of Patient-Reported Outcome Measures Assessing Tumor Visibility and Appearance Concerns in Neurofibromatosis Type 1: A Qualitative Study
This clinical trial update describes the development of patient-reported outcome measures to assess tumor visibility and appearance concerns…
-
CLINICAL_TRIALS research · 2026-04-07Clinical Trial Update: Vismodegib, FAK Inhibitor GSK2256098, Capivasertib, and Abemaciclib in Treating Patients With Progressive Meningiomas [Phase 2]
Phase 2 study evaluating a combination of vismodegib, defactinib, capivasertib, and abemaciclib for intracranial or recurrent meningioma, in…
-
CLINICAL_TRIALS research · 2026-04-06Clinical Trial Update: Evaluation of TQ-B3234 Capsules in Patients With Symptomatic, Non-Surgical Type 1 Neurofibromatosis-Associated Plexiform Neurofibromas [Phase 3]
A Phase 3 clinical trial evaluating TQ-B3234 capsules for symptomatic, non-surgical type 1 neurofibromatosis-associated plexiform neurofibro…
-
CLINICAL_TRIALS research · 2026-03-31Clinical Trial Update: Real-World Treatment Study of Koselugo (Selumetinib)
AstraZeneca is conducting a real-world study of Koselugo (selumetinib) that is currently recruiting for neurofibromatosis type 1 and plexifo…