Nephropathic Cystinosis Market: Analysis of Epidemiology, Pipeline Therapies, and Key Companies Working in the Market

Nephropathic Cystinosis Market: Analysis of Epidemiology, Pipeline Therapies, and Key Companies Working in the Market
DelveInsight Business Research LLP
DelveInsight’s “Nephropathic Cystinosis Market Insights, Epidemiology, and Market Forecast-2032” report delivers an in-depth understanding of the Nephropathic Cystinosis, historical and forecasted epidemiology as well as the Nephropathic Cystinosis market trends in the United States, EU4(Germany, Spain, Italy, France, and United Kingdom) and Japan.

DelveInsight’s “Nephropathic Cystinosis Market Insights, Epidemiology, and Market Forecast-2032” report delivers an in-depth understanding of the Nephropathic Cystinosis, historical and forecasted epidemiology as well as the Nephropathic Cystinosis market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

The Nephropathic Cystinosis market report provides current treatment practices, emerging drugs, Nephropathic Cystinosis market share of the individual therapies, current and forecasted Nephropathic Cystinosis market Size from 2019 to 2032 segmented by seven major markets. The Report also covers current Nephropathic Cystinosis treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the Nephropathic Cystinosis market.

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Nephropathic Cystinosis Overview

Nephropathic cystinosis is a rare autosomal recessive lysosomal storage disorder leading to end-stage renal disease and many extra-renal complications with crystal deposition in the conjunctiva and cornea being the most prominent.

Cystinosis is caused by mutations in the CTNS gene, which encodes the protein cystinosis. Cystinosis is thought to be an H+-driven lysosomal cystine transporter. Genotype–phenotype studies demonstrate that patients with severe nephropathic cystinosis carry mutations that lead to complete loss of cystinosis function. Patients with milder clinical diseases have mutations that lead to the expression of partially functional proteins. Patients with nephropathic cystinosis present with clinical manifestations reflecting their pronounced tubular dysfunction and Fanconi syndrome, including polyuria and polydipsia, growth failure, and rickets. Fever, caused by dehydration or diminished sweat production, is common. Patients are typically fair-skinned and blond because of diminished pigmentation. Ocular presentations include photophobia, retinopathy, and impaired visual acuity. Patients also can develop hypothyroidism, hepatosplenomegaly, and delayed sexual maturation. With progressive tubulointerstitial fibrosis, renal insufficiency is invariant.

 

Nephropathic Cystinosis Epidemiology Insights

Cystinosis is a rare autosomal-recessive lysosomal storage disease with an incidence of ∼0.5–1.0 per 100 000 live births. Although phenotypes may overlap, three clinical forms are recognized. The most severe and frequent form, affecting ∼95% of patients, is termed infantile nephropathic cystinosis (OMIM 219800); patients generally develop clinical symptoms related to renal Fanconi syndrome during the first year of life. Juvenile or late-onset nephropathic cystinosis (OMIM 219900) is usually diagnosed later in childhood or during adolescence; patients present with milder forms of renal Fanconi syndrome or with isolated proteinuria. Finally, the ocular or adult form (OMIM 219750) is characterized by isolated symptoms related to corneal cystine crystal depositions and is rarely diagnosed before adulthood 

 

Nephropathic Cystinosis Epidemiology Segmentation

  • Nephropathic Cystinosis Diagnosed cases 

  • Nephropathic Cystinosis Treatment cases 

  • Nephropathic Cystinosis Incident cases 

  • Nephropathic Cystinosis Prevalent cases 

Nephropathic Cystinosis Market Outlook 

The Nephropathic Cystinosis market outlook of the report helps to build a detailed comprehension of the historic, current, and forecasted Nephropathic Cystinosis market trends by analyzing the impact of current Nephropathic Cystinosis therapies on the market, unmet needs, drivers and barriers, and demand for better technology. This segment gives a thorough detail of the Nephropathic Cystinosis market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated Nephropathic Cystinosis market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

 According to DelveInsight, the Nephropathic Cystinosis market in 7MM is expected to witness a major change in the study period 2019-2032.

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Nephropathic Cystinosis Key Companies 

  • Gilead Sciences 

  • Rocket Pharmaceuticals 

  • Horizon Pharmaceuticals 

  • Centogene Pharmaceutical 

  • And many others 

Nephropathic Cystinosis Therapies

  • Cysteamine bitartrate

  • Cystagon

  • RP103

  • Deferasirox

  • And many others 

Table of Contents

  1.  Key Insights 

  2.  Report Introduction 

  3.  Executive Summary of Nephropathic Cystinosis  

  4.  Disease Background and Overview

  5.  Epidemiology and patient population

  6.  The United States 

  7.  EU 5

  8.  Nephropathic Cystinosis Emerging Therapies

  9.  Nephropathic Cystinosis  Market Outlook

  10.  Market Access and Reimbursement of Therapies

  11.  Market Drivers 

  12.  Market Barriers 

  13.  Appendix

  14.  Nephropathic Cystinosis  Report Methodology

  15.  DelveInsight Capabilities

  16.  Disclaimer

Click here to read more about Nephropathic Cystinosis Market 

Related Reports 

Nephropathic Cystinosis Pipeline Insights, 2023

DelveInsight’s, “Nephropathic Cystinosis (MM) – Pipeline Insights, 2023,” report provides comprehensive insights about 100+ companies and 130+ pipeline drugs in the Nephropathic Cystinosis pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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