Lack of Medication to Drive Neurofibromatosis Therapeutics Pipeline Growth | Top Players are AstraZeneca, Pfizer, GSK, Merck, BioXcel Therapeutics Inc.

Neurofibromatosis is a genetic disorder that affects the nerves throughout the body, resulting in the formation of benign tumors. It is inherited in an autosomal-dominant manner, meaning that it is not necessary that the mutated gene is passed on from both the parents. The manifestation of the disorder varies from individual to individual. There are two major types of neurofibromatosis — neurofibromatosis type I (NF-1) and neurofibromatosis type I (NF-2) —, and a third type has now been recognized, named Schwannomatosis.

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NF-1is caused by mutations in the NF-1 gene, which is responsible for neurofibromin production in the body. Neurofibromin is a protein that is produced in many cells, including oligodendrocytes and Schwann cells. It functions as a tumor suppressor by keeping cell proliferation in check. Mutations in the NF-1 gene cause neurofibromin to lose its tumor suppressor function, resulting in the formation of neurofibromas (benign tumors that grow on the nerves).

NF-1, which manifests after birth or during early childhood, is characterized by the presence of benign tumors under the skin and multiple café-au-lait (coffee with milk) spots around the underarms and groin region. Scoliosis and enlargement and deformity of the bones may also be present. Instances of tumor development may also be seen on the cranial nerves, in the brain, and spinal cord. Occasionally, vision problems, speech impairment, and behavioral issues might also be reported.

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Similarly, mutations in the NF-2 gene, responsible for the synthesis of a protein named merlin or Schwannomin, cause NF-2. Schwann cells, which surround and insulate the neurons present in the brain and spinal cord, produce this protein. It is also a tumor-suppressor protein, working on the same lines as neurofibromin. Its functional loss may result in uncontrolled cell multiplication, especially of Schwann cells, resulting in tumor formation. NF-2 may affect people during childhood, adolescence or early adulthood. It affects the nerves that transmit audio signals from the inner ear to the brain.

Schwannomatosis is more prevalent in adults older than 30 years. It may share many characteristics with NF type 1 and 2, but the reported evidence suggests it to be a distinct condition. There is no treatment available for NF yet, and it is done on the basis of the symptoms that appear. Surgery is one of the ways to relieve pain and improve neurologic functions, and also for diagnostic purposes. Chemotherapy or radiation is used for destroying rapidly proliferating cells and shrink the neurofibromas. Few chemotherapy drugs are vinblastine, doxorubicin, carboplatin, methotrexate, and ifosfamide.

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One of the driving factors for the neurofibromatosis therapeutics pipeline is the lack of medication. Scientists are trying to find distinct biomarkers for NF-1, NF-2, and Schwannomatosis, as even though these have similar symptoms, they are genetically distinct. They are used for either predictive, prognosis, or pharmacodynamic purposes. Many institutes, such as the New York University School of Medicine, University of Texas Health Science Center, and Indiana University, are collaborating with pharmaceutical companies to improve the scenario of biomarker discovery and drug development.

There were 15 drugs in the Phase II development phase of the neurofibromatosis therapeutics pipeline as of February 2018. One of the candidates is selumetinib, a small molecule with potential antineoplastic activity, which is being pursued by AstraZeneca plc. This molecule inhibits the activity of protein kinase 1 and 2 in an adenosine triphosphate-independent manner. Besides neurofibromatosis, it is being investigated for the treatment for AIDS-related Kaposi's sarcoma, optic nerve glioma, meningioma, vestibular Schwannoma, and uveal melanoma.

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This release was published on openPR.