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NF1-PN

NF1-PN
Specialty Channel
Angela Hirbe, MD, PhD
Videos
Mirdametinib Demonstrates Durable Responses in NF1-Associated Plexiform Neurofibromas
04/14/2026
Angela Hirbe, MD, PhD
Angela Hirbe, MD, PhD, discusses long-term follow-up results from the phase 2b ReNeu trial evaluating mirdametinib among adult and pediatric patients with inoperable, symptomatic NF1-associated plexiform neurofibromas.
Angela Hirbe, MD, PhD, discusses long-term follow-up results from the phase 2b ReNeu trial evaluating mirdametinib among adult and pediatric patients with inoperable, symptomatic NF1-associated plexiform neurofibromas.
Angela Hirbe, MD, PhD, discusses...
04/14/2026
Oncology
FDA Approval
FDA Grants Approval to Selumetinib for Adults With NF1 and Symptomatic, Inoperable Plexiform Neurofibromas
11/19/2025
Emily Estrada
On November 19, 2025, the FDA approved selumetinib for adults with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas, expanding its prior pediatric indication to adults based on results from the phase 3 KOMET trial.
On November 19, 2025, the FDA approved selumetinib for adults with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas, expanding its prior pediatric indication to adults based on results from the phase 3 KOMET trial.
On November 19, 2025, the FDA...
11/19/2025
Oncology
Angela Hirbe, MD, PhD; Julia Meade, MD; Carlos Romo, MD
Videos
Future Therapeutic Options in Neurofibromatosis Type 1 With Plexiform Neurofibromas
04/29/2025
Angela Hirbe, MD, PhD; Carlos Romo, MD; Julia Meade, MD
In this video, a panel of experts discuss the management of patients with neurofibromatosis type 1 with plexiform neurofibromas, including future research areas.
In this video, a panel of experts discuss the management of patients with neurofibromatosis type 1 with plexiform neurofibromas, including future research areas.
In this video, a panel of...
04/29/2025
Oncology
Angela Hirbe, MD, PhD; Julia Meade, MD; Carlos Romo, MD
Videos
Mirdametinib for Adult and Pediatric Patients With Neurofibromatosis Type 1 With Plexiform Neurofibromas
04/29/2025
Angela Hirbe, MD, PhD; Carlos Romo, MD; Julia Meade, MD
In this video, a panel of experts discuss the management of patients with neurofibromatosis type 1 with plexiform neurofibromas, including data from the ReNeu trial.
In this video, a panel of experts discuss the management of patients with neurofibromatosis type 1 with plexiform neurofibromas, including data from the ReNeu trial.
In this video, a panel of...
04/29/2025
Oncology
Angela Hirbe, MD, PhD; Julia Meade, MD; Carlos Romo, MD
Videos
Potential Therapeutic Options for Patients With Neurofibromatosis Type 1 With Plexiform Neurofibromas
04/29/2025
Angela Hirbe, MD, PhD; Carlos Romo, MD; Julia Meade, MD
In this video, a panel of experts discuss the management of patients with neurofibromatosis type 1 with plexiform neurofibromas, including the potential therapeutic options.
In this video, a panel of experts discuss the management of patients with neurofibromatosis type 1 with plexiform neurofibromas, including the potential therapeutic options.
In this video, a panel of...
04/29/2025
Oncology
News
Mirdametinib for Adult and Pediatric Patients With Neurofibromatosis Type 1 With Plexiform Neurofibromas
11/12/2024
Allison Casey
According to a phase 2b trial, mirdametinib demonstrated significant confirmed objective response rates with deep and durable reductions of volume of target plexiform neurofibromas and clinically meaningful improvements to health-related...
According to a phase 2b trial, mirdametinib demonstrated significant confirmed objective response rates with deep and durable reductions of volume of target plexiform neurofibromas and clinically meaningful improvements to health-related...
According to a phase 2b trial,...
11/12/2024
Oncology
News
Management of Adverse Events Associated With Selumetinib for Pediatric Patients With Neurofibromatosis Type 1 With Plexiform Neurofibromas
09/17/2024
Allison Casey
A panel of experts provided consensus recommendations on prevention, early detection, and management of adverse events potentially associated with selumetinib among pediatric patients with neurofibromatosis type 1 and plexiform neurofibromas.
A panel of experts provided consensus recommendations on prevention, early detection, and management of adverse events potentially associated with selumetinib among pediatric patients with neurofibromatosis type 1 and plexiform neurofibromas.
A panel of experts provided...
09/17/2024
Oncology
News
Hearing Loss Among Pediatric Patients with Neurofibromatosis Type 1
08/29/2024
Allison Casey
According to a review of audiometric data, the rate of hearing loss among patients with neurofibromatosis type 1 (NF1) compared to the general population was higher than expected.
According to a review of audiometric data, the rate of hearing loss among patients with neurofibromatosis type 1 (NF1) compared to the general population was higher than expected.
According to a review of...
08/29/2024
Oncology
News
Effect of Food on Selumetinib Dosing for Adolescent Patients With Neurofibromatosis Type 1 With Plexiform Neurofibromas
08/01/2024
Allison Casey
In the phase 1 trial, the addition of a low-fat meal with doses of selumetinib did not significantly impact selumetinib exposure or gastrointestinal toxicities among adolescent patients with neurofibromatosis type 1 with plexiform...
In the phase 1 trial, the addition of a low-fat meal with doses of selumetinib did not significantly impact selumetinib exposure or gastrointestinal toxicities among adolescent patients with neurofibromatosis type 1 with plexiform...
In the phase 1 trial, the...
08/01/2024
Oncology
News
Selumetinib Shows Efficacy, Safety Among Pediatric and Adult Patients With Neurofibromatosis Type 1 and Plexiform Neurofibroma
07/09/2024
Allison Casey
According to a phase 2 study, selumetinib demonstrated efficacy among both children and adults with NF1 and inoperable plexiform neurofibromas, and in other manifestations.
According to a phase 2 study, selumetinib demonstrated efficacy among both children and adults with NF1 and inoperable plexiform neurofibromas, and in other manifestations.
According to a phase 2 study,...
07/09/2024
Oncology
News
Study Findings Show Considerable Disease Burden of NF1-PN Among Affected Patients
07/01/2024
Janelle Bradley
Findings from the real-world CASSIOPEA study show the considerable disease burden of neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN) highlighting the importance of effective treatments for this patient population.
Findings from the real-world CASSIOPEA study show the considerable disease burden of neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN) highlighting the importance of effective treatments for this patient population.
Findings from the real-world...
07/01/2024
Oncology

News

FDA Approval
FDA Grants Approval to Selumetinib for Adults With NF1 and Symptomatic, Inoperable Plexiform Neurofibromas
11/19/2025
Emily Estrada
On November 19, 2025, the FDA approved selumetinib for adults with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas, expanding its prior pediatric indication to adults based on results from the phase 3 KOMET trial.
On November 19, 2025, the FDA approved selumetinib for adults with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas, expanding its prior pediatric indication to adults based on results from the phase 3 KOMET trial.
On November 19, 2025, the FDA...
11/19/2025
Oncology
News
Mirdametinib for Adult and Pediatric Patients With Neurofibromatosis Type 1 With Plexiform Neurofibromas
11/12/2024
Allison Casey
According to a phase 2b trial, mirdametinib demonstrated significant confirmed objective response rates with deep and durable reductions of volume of target plexiform neurofibromas and clinically meaningful improvements to health-related...
According to a phase 2b trial, mirdametinib demonstrated significant confirmed objective response rates with deep and durable reductions of volume of target plexiform neurofibromas and clinically meaningful improvements to health-related...
According to a phase 2b trial,...
11/12/2024
Oncology
News
Management of Adverse Events Associated With Selumetinib for Pediatric Patients With Neurofibromatosis Type 1 With Plexiform Neurofibromas
09/17/2024
Allison Casey
A panel of experts provided consensus recommendations on prevention, early detection, and management of adverse events potentially associated with selumetinib among pediatric patients with neurofibromatosis type 1 and plexiform neurofibromas.
A panel of experts provided consensus recommendations on prevention, early detection, and management of adverse events potentially associated with selumetinib among pediatric patients with neurofibromatosis type 1 and plexiform neurofibromas.
A panel of experts provided...
09/17/2024
Oncology
News
Hearing Loss Among Pediatric Patients with Neurofibromatosis Type 1
08/29/2024
Allison Casey
According to a review of audiometric data, the rate of hearing loss among patients with neurofibromatosis type 1 (NF1) compared to the general population was higher than expected.
According to a review of audiometric data, the rate of hearing loss among patients with neurofibromatosis type 1 (NF1) compared to the general population was higher than expected.
According to a review of...
08/29/2024
Oncology
News
Effect of Food on Selumetinib Dosing for Adolescent Patients With Neurofibromatosis Type 1 With Plexiform Neurofibromas
08/01/2024
Allison Casey
In the phase 1 trial, the addition of a low-fat meal with doses of selumetinib did not significantly impact selumetinib exposure or gastrointestinal toxicities among adolescent patients with neurofibromatosis type 1 with plexiform...
In the phase 1 trial, the addition of a low-fat meal with doses of selumetinib did not significantly impact selumetinib exposure or gastrointestinal toxicities among adolescent patients with neurofibromatosis type 1 with plexiform...
In the phase 1 trial, the...
08/01/2024
Oncology
News
Selumetinib Shows Efficacy, Safety Among Pediatric and Adult Patients With Neurofibromatosis Type 1 and Plexiform Neurofibroma
07/09/2024
Allison Casey
According to a phase 2 study, selumetinib demonstrated efficacy among both children and adults with NF1 and inoperable plexiform neurofibromas, and in other manifestations.
According to a phase 2 study, selumetinib demonstrated efficacy among both children and adults with NF1 and inoperable plexiform neurofibromas, and in other manifestations.
According to a phase 2 study,...
07/09/2024
Oncology
News
Study Findings Show Considerable Disease Burden of NF1-PN Among Affected Patients
07/01/2024
Janelle Bradley
Findings from the real-world CASSIOPEA study show the considerable disease burden of neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN) highlighting the importance of effective treatments for this patient population.
Findings from the real-world CASSIOPEA study show the considerable disease burden of neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN) highlighting the importance of effective treatments for this patient population.
Findings from the real-world...
07/01/2024
Oncology
News
Vinblastine/Methotrexate Fails to Demonstrate Activity for NF1-PN in Pediatric and Young-Adult Patients
07/01/2024
Janelle Bradley
Findings from a phase 2 study show vinblastine/methotrexate did not demonstrate activity for neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN) in pediatric and young-adult patients, despite being well-tolerated.
Findings from a phase 2 study show vinblastine/methotrexate did not demonstrate activity for neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN) in pediatric and young-adult patients, despite being well-tolerated.
Findings from a phase 2 study...
07/01/2024
Oncology
News
Health-Related Quality of Life Among Adult Patients With NF1-PN and Caregivers
07/01/2024
Janelle Bradley
Study findings show neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN) worsens adult patient and caregiver health-related quality of life vs that of the general population.
Study findings show neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN) worsens adult patient and caregiver health-related quality of life vs that of the general population.
Study findings show...
07/01/2024
Oncology
News
Selumetinib for Managing NF1-PN in Pediatric Patients at Risk for Developing Tumor-Related Morbidity
07/01/2024
Janelle Bradley
Among pediatric patients with neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN), treatment with selumetinib resulted in significant shrinkage in PN without any clinically significant morbidity.
Among pediatric patients with neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN), treatment with selumetinib resulted in significant shrinkage in PN without any clinically significant morbidity.
Among pediatric patients with...
07/01/2024
Oncology

Interactive Features

Recent News

FDA Approval
FDA Grants Approval to Selumetinib for Adults With NF1 and Symptomatic, Inoperable Plexiform Neurofibromas
11/19/2025
Emily Estrada
On November 19, 2025, the FDA approved selumetinib for adults with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas, expanding its prior pediatric indication to adults based on results from the phase 3 KOMET trial.
On November 19, 2025, the FDA approved selumetinib for adults with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas, expanding its prior pediatric indication to adults based on results from the phase 3 KOMET trial.
On November 19, 2025, the FDA...
11/19/2025
Oncology
News
Mirdametinib for Adult and Pediatric Patients With Neurofibromatosis Type 1 With Plexiform Neurofibromas
11/12/2024
Allison Casey
According to a phase 2b trial, mirdametinib demonstrated significant confirmed objective response rates with deep and durable reductions of volume of target plexiform neurofibromas and clinically meaningful improvements to health-related...
According to a phase 2b trial, mirdametinib demonstrated significant confirmed objective response rates with deep and durable reductions of volume of target plexiform neurofibromas and clinically meaningful improvements to health-related...
According to a phase 2b trial,...
11/12/2024
Oncology
News
Management of Adverse Events Associated With Selumetinib for Pediatric Patients With Neurofibromatosis Type 1 With Plexiform Neurofibromas
09/17/2024
Allison Casey
A panel of experts provided consensus recommendations on prevention, early detection, and management of adverse events potentially associated with selumetinib among pediatric patients with neurofibromatosis type 1 and plexiform neurofibromas.
A panel of experts provided consensus recommendations on prevention, early detection, and management of adverse events potentially associated with selumetinib among pediatric patients with neurofibromatosis type 1 and plexiform neurofibromas.
A panel of experts provided...
09/17/2024
Oncology
News
Hearing Loss Among Pediatric Patients with Neurofibromatosis Type 1
08/29/2024
Allison Casey
According to a review of audiometric data, the rate of hearing loss among patients with neurofibromatosis type 1 (NF1) compared to the general population was higher than expected.
According to a review of audiometric data, the rate of hearing loss among patients with neurofibromatosis type 1 (NF1) compared to the general population was higher than expected.
According to a review of...
08/29/2024
Oncology
News
Effect of Food on Selumetinib Dosing for Adolescent Patients With Neurofibromatosis Type 1 With Plexiform Neurofibromas
08/01/2024
Allison Casey
In the phase 1 trial, the addition of a low-fat meal with doses of selumetinib did not significantly impact selumetinib exposure or gastrointestinal toxicities among adolescent patients with neurofibromatosis type 1 with plexiform...
In the phase 1 trial, the addition of a low-fat meal with doses of selumetinib did not significantly impact selumetinib exposure or gastrointestinal toxicities among adolescent patients with neurofibromatosis type 1 with plexiform...
In the phase 1 trial, the...
08/01/2024
Oncology
News
Selumetinib Shows Efficacy, Safety Among Pediatric and Adult Patients With Neurofibromatosis Type 1 and Plexiform Neurofibroma
07/09/2024
Allison Casey
According to a phase 2 study, selumetinib demonstrated efficacy among both children and adults with NF1 and inoperable plexiform neurofibromas, and in other manifestations.
According to a phase 2 study, selumetinib demonstrated efficacy among both children and adults with NF1 and inoperable plexiform neurofibromas, and in other manifestations.
According to a phase 2 study,...
07/09/2024
Oncology
News
Study Findings Show Considerable Disease Burden of NF1-PN Among Affected Patients
07/01/2024
Janelle Bradley
Findings from the real-world CASSIOPEA study show the considerable disease burden of neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN) highlighting the importance of effective treatments for this patient population.
Findings from the real-world CASSIOPEA study show the considerable disease burden of neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN) highlighting the importance of effective treatments for this patient population.
Findings from the real-world...
07/01/2024
Oncology
News
Vinblastine/Methotrexate Fails to Demonstrate Activity for NF1-PN in Pediatric and Young-Adult Patients
07/01/2024
Janelle Bradley
Findings from a phase 2 study show vinblastine/methotrexate did not demonstrate activity for neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN) in pediatric and young-adult patients, despite being well-tolerated.
Findings from a phase 2 study show vinblastine/methotrexate did not demonstrate activity for neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN) in pediatric and young-adult patients, despite being well-tolerated.
Findings from a phase 2 study...
07/01/2024
Oncology
News
Health-Related Quality of Life Among Adult Patients With NF1-PN and Caregivers
07/01/2024
Janelle Bradley
Study findings show neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN) worsens adult patient and caregiver health-related quality of life vs that of the general population.
Study findings show neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN) worsens adult patient and caregiver health-related quality of life vs that of the general population.
Study findings show...
07/01/2024
Oncology
News
Selumetinib for Managing NF1-PN in Pediatric Patients at Risk for Developing Tumor-Related Morbidity
07/01/2024
Janelle Bradley
Among pediatric patients with neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN), treatment with selumetinib resulted in significant shrinkage in PN without any clinically significant morbidity.
Among pediatric patients with neurofibromatosis type 1 with plexiform neurofibromas (NF1-PN), treatment with selumetinib resulted in significant shrinkage in PN without any clinically significant morbidity.
Among pediatric patients with...
07/01/2024
Oncology
News
Camrelizumab Plus Apatinib Demonstrates Encoruaging Activity in Patients With Refractory Chordoma
06/26/2026
Emily Estrada
According to results from an investigator-initiated phase 2 study, camrelizumab plus apatinib demonstrated encouraging efficacy and durable disease control in patients with refractory chordoma.
According to results from an investigator-initiated phase 2 study, camrelizumab plus apatinib demonstrated encouraging efficacy and durable disease control in patients with refractory chordoma.
According to results from an...
06/26/2026
Oncology
News
Ten-Year Follow-Up Supports Curative Potential of Tisagenlecleucel in Patients With B-Cell Non-Hodgkin Lymphomas
06/25/2026
Stephanie Holland
Long-term follow-up of patients with relapsed or refractory B-cell non-Hodgkin lymphoma demonstrated that tisagenlecleucel induced durable remissions, with no disease relapses occurring beyond 5.4 years after treatment.
Long-term follow-up of patients with relapsed or refractory B-cell non-Hodgkin lymphoma demonstrated that tisagenlecleucel induced durable remissions, with no disease relapses occurring beyond 5.4 years after treatment.
Long-term follow-up of patients...
06/25/2026
Oncology
News
Pirtobrutinib Demonstrates Durable Clinical Activity in Relapsed or Refractory Waldenström Macroglobulinemia
06/25/2026
Stephanie Holland
Results from the phase 1/2 BRUIN trial demonstrated that pirtobrutinib shows durable clinical activity in relapsed or refractory Waldenström macroglobulinemia, including those previously treated with covalent Bruton tyrosine kinase inhibitors.
Results from the phase 1/2 BRUIN trial demonstrated that pirtobrutinib shows durable clinical activity in relapsed or refractory Waldenström macroglobulinemia, including those previously treated with covalent Bruton tyrosine kinase inhibitors.
Results from the phase 1/2 BRUIN...
06/25/2026
Oncology
News
Nivolumab Plus Ipilimumab Shows Encouraging Activity in Advanced Gallbladder Carcinoma
06/25/2026
Stephanie Holland
According to results from the phase 2 MoST-CIRCUIT trial, nivolumab plus ipilimumab demonstrated encouraging activity in patients with gallbladder carcinoma despite limited overall efficacy in patients with advanced biliary tract cancers.
According to results from the phase 2 MoST-CIRCUIT trial, nivolumab plus ipilimumab demonstrated encouraging activity in patients with gallbladder carcinoma despite limited overall efficacy in patients with advanced biliary tract cancers.
According to results from the...
06/25/2026
Oncology
News
Neoadjuvant Nivolumab Demonstrates Strong Activity in Resectable Mismatch Repair-Deficient Endometrial Cancer
06/23/2026
Emily Estrada
Results from a phase 2 study demonstrated that neoadjuvant nivolumab induced high rates of complete response among patients with resectable mismatch repair-deficient endometrial cancer.
Results from a phase 2 study demonstrated that neoadjuvant nivolumab induced high rates of complete response among patients with resectable mismatch repair-deficient endometrial cancer.
Results from a phase 2 study...
06/23/2026
Oncology
News
Bulumtatug Fuvedotin Demonstrates Durable Activity in Previously Treated Cervical Cancer
06/23/2026
Emily Estrada
Updated results from a phase 2 study demonstrated that bulumtatug fuvedotin shows encouraging efficacy and manageable tolerability in patients with recurrent or metastatic cervical cancer, including those previously treated with immune...
Updated results from a phase 2 study demonstrated that bulumtatug fuvedotin shows encouraging efficacy and manageable tolerability in patients with recurrent or metastatic cervical cancer, including those previously treated with immune...
Updated results from a phase 2...
06/23/2026
Oncology
FDA Approval
FDA Approves Afamitresgene Autoleucel for Patients With Unresectable or Metastatic Synovial Sarcoma
06/23/2026
Stephanie Holland
Based on results from the SPEARHEAD-1 study, the FDA granted full approval to afamitresgene autoleucel for unresectable or metastatic synovial sarcoma and expanded its indication to include eligible patients aged 12 years and older.
Based on results from the SPEARHEAD-1 study, the FDA granted full approval to afamitresgene autoleucel for unresectable or metastatic synovial sarcoma and expanded its indication to include eligible patients aged 12 years and older.
Based on results from the...
06/23/2026
Oncology
News
Signatera-Based ctDNA Testing Incorporated Into NCCN Bladder Cancer Guidelines
06/23/2026
Stephanie Holland
The NCCN has updated its Clinical Practice Guidelines in Oncology for Bladder Cancer to include tumor-informed ctDNA-MRD testing as a tool for risk stratification and treatment selection in patients with muscle-invasive bladder cancer.
The NCCN has updated its Clinical Practice Guidelines in Oncology for Bladder Cancer to include tumor-informed ctDNA-MRD testing as a tool for risk stratification and treatment selection in patients with muscle-invasive bladder cancer.
The NCCN has updated its...
06/23/2026
Oncology
News
Dostarlimab Demonstrates Durable 6-Year Survival Benefit in dMMR/MSI-H Endometrial Cancer
06/18/2026
Stephanie Holland
Updated results from the phase 1 GARNET trial demonstrated durable long-term clinical benefit with dostarlimab monotherapy in patients with dMMR/MSI-H advanced or recurrent endometrial cancer.
Updated results from the phase 1 GARNET trial demonstrated durable long-term clinical benefit with dostarlimab monotherapy in patients with dMMR/MSI-H advanced or recurrent endometrial cancer.
Updated results from the phase 1...
06/18/2026
Oncology
News
Updated Results from the RUBY Trial Reinforce Dostarlimab as Standard of Care in dMMR/MSI-H Endometrial Cancer
06/18/2026
Stephanie Holland
Updated results from the phase 3 RUBY trial demonstrated that dostarlimab plus carboplatin and paclitaxel sustained survival benefit in patients with dMMR/MSI-H primary advanced or recurrent endometrial cancer.
Updated results from the phase 3 RUBY trial demonstrated that dostarlimab plus carboplatin and paclitaxel sustained survival benefit in patients with dMMR/MSI-H primary advanced or recurrent endometrial cancer.
Updated results from the phase 3...
06/18/2026
Oncology

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