Amyloidosis is a rare disease characterized by an abnormal accumulation of a protein called amyloid throughout the body. Chemotherapy treatments, cardiac medications, targeted therapies, organ transplant, dialysis, and autologous blood stem cell transplant are all utilized to treat amyloidosis. Amyloid light chain amyloidosis, autoimmune amyloidosis, familial amyloidosis, wide-type (or senile) amyloidosis, dialysis-related, and cardiac amyloidosis are the most prevalent forms of amyloidosis.
The aggregation and deposition of mutant and wild-type transthyretin (TTR) protein in the heart characterizes familial amyloid cardiomyopathy (FAC) or transthyretin amyloid cardiomyopathy (ATTR-CM). The nerves, heart, and kidneys are frequently affected by this hereditary disease. FAC may have an impact on how electrical signals go through the heart (conduction system). This can result in arrhythmias (abnormal heartbeats) and incorrect cardiac signals. It has a dismal prognosis, with a median survival time of about 13 months without therapy. The frequent TTR gene mutation known as Val30Met, which is related with familial transthyretin amyloidosis (FTA), affects around 1 in 583 individuals in some regions of Portugal, compared to approximately 1 in 100,000 people in the United States. Treatment for familial amyloid cardiomyopathy is expensive and restricted. There is no therapy for FAC that can reverse the harm caused by amyloid accumulation.
Treatment for primary familial amyloid cardiomyopathy includes liver transplantation. Heart and/or kidney transplantation, replacement of the liquid component of the eye (vitrectomy) for ocular involvement, and carpal tunnel surgery are all possible therapies. Patisiran (Onpattro) and Inotersen are two FDA-approved therapies for familial amyloid cardiomyopathy (Tegsedi). Patisiran was authorized in August 2018 for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, and Inotersen was approved in October 2018 for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. Patisiran is a double-stranded short interfering RNA that specifically targets a region in the transthyretin (TTR) messenger RNA. Depending on the pharmacy, Onpattro intravenous solution (2 mg/mL) costs about US$ 9,928 for a supply of 5 milliliters. Alnylam Pharmaceuticals, Inc., an RNAi therapeutics company, announced positive results from a Phase 3b open-label study to evaluate the safety, efficacy, and pharmacokinetics of patisiran in patients with hereditary ATTR (hATTR) amyloidosis who progressed to polyneuropathy after receiving an orthotopic liver transplant in June 2021.
Inotersen is a 2-MOE antisense oligonucleotide drug that reduces the quantity of mutant and normal TTR produced by the liver. Ionis Pharmaceuticals, Inc., an RNA-targeted therapy developer, concluded enrollment in NEURO-TTRansform, the company's Phase 3 trial of eplontersen for patients with hereditary transthyretin-mediated amyloid polyneuropathy, in July 2021. (hATTR-PN). The findings will be compared to those of the inotersen NEURO-TTR registrational trial, which was completed in 2017.
Several businesses are working to find viable treatments for familial amyloid cardiomyopathy. Novo Nordisk purchased Prothena, a late-stage clinical firm, in July 2021 for clinical-stage antibody PRX004, an experimental treatment for patients with hereditary transthyretin amyloidosis. The purchase is intended to help develop PRX004 for the treatment of ATTR cardiomyopathy. PRX004 is intended to remove transthyretin misfolded forms.
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The major players in the familial amyloid polyneuropathy therapeutics market include Pfizer Inc., Alnylam Pharmaceuticals Inc., Ionis Pharmaceuticals Inc., Corino Therapeutics Inc., Proclara Biosciences, Arcturus Therapeutics Inc.
This section includes a holistic view of the competitive landscape that includes various strategic developments such as key mergers & acquisitions, future capacities, partnerships, financial overviews, collaborations, new product developments, new product launches, and other developments.Get more information on "Global Familial Amyloid Polyneuropathy Therapeutics Market Research Report" by requesting FREE Sample Copy at https://www.valuemarketresearch.com/contact/familial-amyloid-polyneuropathy-therapeutics-market/download-sampleMarket DynamicsRising awareness about FAP, a multi-visceral and life-threatening disease resulting in multiple disabilities, is the primary stimulant for this growth market.
The development of innovative therapies and increased adoption of therapeutic drugs to treat this progressive disease affecting the peripheral and autonomic nervous system will boost the market growth.
The multidisciplinary approach used in treating FAP will serve as a boon for this market's steady growth.
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The major players in the familial amyloid polyneuropathy therapeutics market include Pfizer Inc., Alnylam Pharmaceuticals Inc., Ionis Pharmaceuticals Inc., Corino Therapeutics Inc., Proclara Biosciences, Arcturus Therapeutics Inc.
This section includes a holistic view of the competitive landscape that includes various strategic developments such as key mergers & acquisitions, future capacities, partnerships, financial overviews, collaborations, new product developments, new product launches, and other developments.This section covers regional segmentation which accentuates on current and future demand for familial amyloid polyneuropathy therapeutics market across North America, Europe, Asia-Pacific, Latin America, and Middle East & Africa.
Further, the report focuses on demand for individual application segment across all the prominent regions.Purchase complete Global Familial Amyloid Polyneuropathy Therapeutics Market Research Report At: https://www.valuemarketresearch.com/contact/familial-amyloid-polyneuropathy-therapeutics-market/buy-nowAbout Us:Value Market Research was established with the vision to ease decision making and empower the strategists by providing them with holistic market information.We facilitate clients with syndicate research reports and customized research reports on 25+ industries with global as well as regional coverage.Contact:Value Market Research401/402, TFM, Nagras Road, Aundh, Pune-7.Maharashtra, INDIA.Tel: +1-888-294-1147Email: [email protected]: https://www.valuemarketresearch.com
