FDA Approves Koselugo (selumetinib) for Pediatric Patients with Neurofibromatosis Type 1 Plexiform Neurofibromas

13 April 2020 -- AstraZeneca and MSD Inc., Kenilworth, N.J., US (MSD: known as Merck&Co., Inc. inside the US and Canada) today announced that the US Food and Drug Administration (FDA) has approved the kinase inhibitor Koselugo (selumetinib)...
Source: Drugs.com - New Drug Approvals - Category: Drugs & Pharmacology Source Type: news

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ConclusionsAlthough SPECT imaging still represents the gold standard for CBF assessment, our results suggest that data obtained using DSC perfusion technique, and in particular MTT maps, might be a very useful and noninvasive tool for evaluating hemodynamic status in MMS-NF1 patients.
Source: Neurological Sciences - Category: Neurology Source Type: research
Conditions:   Prader-Willi Syndrome;   PWS-like Syndrome;   Silver Russel Syndrome;   Congenital Hypopituitarism;   Klinefelter (XXY-)Syndrome;   Congenital Adrenal Hyperplasia;   XXXXY Syndrome;   XXYY Syndrome;   XXXX Syndrome (Tetra-X Syndrome);   Disorders of Sex Development;   T urner Syndrome;   46, XY DSD;   Tuberous Sclerosis;   Neurofibromatosis;   Albright Hereditaire Osteodystrofie;   Cornelia de Lange Syndrome...
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Condition:   Neurofibromatosis Intervention:   Drug: NPC-12G gel Sponsor:   Nobelpharma Recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
uan Bai Patients with RASopathy Neurofibromatosis 1 (NF1) are at a markedly increased risk of the development of benign and malignant tumors. Malignant tumors are often recalcitrant to treatments and associated with poor survival; however, no chemopreventative strategies currently exist. We thus evaluated the effect of mebendazole, alone or in combination with cyclooxygenase-2 (COX-2) inhibitors, on the prevention of NF1-related malignancies in a cis Nf1+/-;Tp53+/- (NPcis) mouse model of NF1. Our in vitro findings showed that mebendazole (MBZ) inhibits the growth of NF1-related malignant peripheral nerve sheath tumors ...
Source: Genes - Category: Genetics & Stem Cells Authors: Tags: Article Source Type: research
ConclusionClinicians must consider the possibility of soft tissue sarcoma even in a patient with a small, slow-growing, superficial mass. Furthermore, a wrong open biopsy or nononcological surgical procedure may lead to possible contamination and ultimately a more radical procedure than would have originally been necessary, where this can be prevented by an early referral to a highly specialized sarcoma center.
Source: Journal of Medical Case Reports - Category: General Medicine Source Type: research
Conclusion: To our knowledge, this case is the fifth report of pineal PXA and the first associated with NF1. Because PXA presents similarly to other NF1-related intracranial tumors, careful diagnosis via immunohistochemistry is imperative. Gross tumor resection is usually curative; however, PXA has the propensity to undergo malignant degeneration and may require adjuvant treatment. PMID: 32612482 [PubMed]
Source: Ochsner Journal - Category: General Medicine Tags: Ochsner J Source Type: research
Neurofibromatoses (NF) are a group of genetically distinct disorders of the nervous system unified by the predisposition to nerve sheath tumors. Although adolescents with NF types 1 and 2 (NF1 and NF2) report poor quality of life and high psychosocial burden, there are no evidence-based interventions to address these needs. This paper presents the study design and protocol for the first randomized controlled trial (RCT) of a mind-body intervention for adolescents with NF, Resilient Youth with NF (RY-NF), versus an educational control group, Health Education for NF (HE-NF), both delivered in groups via secure live video.
Source: Contemporary Clinical Trials - Category: Radiology Authors: Source Type: research
CONCLUSION: Our case illustrates the complexities of multiple genetic mutations in a child. PMID: 32597698 [PubMed - as supplied by publisher]
Source: Fetal and Pediatric Pathology - Category: Pathology Tags: Fetal Pediatr Pathol Source Type: research
Rationale: Malignant peripheral nerve sheath tumor (MPNST) is a rare sarcoma. Owing to the lack of specific histological criteria, immunohistochemical, and molecular diagnostic markers, several differential diagnoses must be considered. Advances in molecular testing can provide significant insights for management of rare tumor. Patient concerns: The patient was a 50-year-old man with a history of lumpectomy on the right back 30 years ago. He felt a stabbing pain at the right iliac fossa and went to the local hospital. Diagnosis: By immunohistochemistry, the tumor cells stained positively for S-100 (focal +), CD34 (...
Source: Medicine - Category: Internal Medicine Tags: Research Article: Clinical Case Report Source Type: research
Neurofibromatosis type 2 (NF2) is a rare genetic disease involving multiple tumors of the central and peripheral nervous systems. Most patients with NF2 have bilateral vestibular schwannomas; nonvestibular sch...
Source: Journal of Otolaryngology - Head and Neck Surgery - Category: ENT & OMF Authors: Tags: Case report Source Type: research
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