DelveInsight’s “Phenylketonuria (PKU) Market Insights Report” offers a detailed analysis of the disease, its causes, symptoms, diagnostics, modalities, and treatment options.
The Phenylketonuria market report also covers Phenylketonuria market size, epidemiology, developing medicines, market drivers, market hurdles, ongoing clinical studies, significant partnerships in the sector, and key pharmaceutical companies actively driving the Phenylketonuria market size forward.
Some of the key highlights from the Phenylketonuria Market Insights:
- The overall number of diagnosed Phenylketonuria patients in the 7MM was 49,887 in 2020, and this number is expected to rise by 2030, with a CAGR of 0.58 percent for the study period 2018–2030.
- In the 7MM, there were 24,164 cases of Classic Phenylketonuria (cPKU), 14,752 cases of Mild Phenylketonuria (mPKU), and 10,971 cases of Mild hyperphenylalaninemia (MHP)in 2020. In comparison to the other 7MM countries, the United States has the highest number of cases of Phenylketonuria.
- Germany had the most number of cases, with 8,317, followed by France with 7,523 and the United Kingdom with 6,564. With 2,490 instances in 2020, Spain had the fewest cases.
- The expected launch of emerging therapies with unique mechanisms of action such as SYNB-1618 and CNSA-001 shall impact the Phenylketonuria market size in upcoming years.
- SYNB-1618 is the most anticipated emerging therapy, awaiting launch. Apart from this, other products include CNSA-001, BMN 307, and one gene therapy HMI-102. Some of these products have also received Fastrack and Orphan Designations.
- Companies working on therapies to treat and manage Phenylketonuria include Synlogic, Censa Pharmaceuticals, Homology Medicines, Biomarin Pharmaceuticals, and others. These therapies are currently in various stages of development.
- Key Phenylketonuria pipeline therapies include SYNB1618, CNSA-001, HMI-102, BMN 307 among others.
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Phenylketonuria is a metabolic disorder that can be detected during the first few days of life through regular newborn screening. The enzyme phenylalanine hydroxylase (PAH), which is responsible for digesting the amino acid phenylalanine, is absent or deficient in Phenylketonuria.
Proteins’ chemical building blocks, amino acids, are required for normal growth and development. Phenylalanine is metabolized to tyrosine in the presence of normal PAH activity. When PAH is missing or insufficient, phenylalanine builds up and becomes toxic to the brain.
Most patients with Phenylketonuria would have a serious intellectual handicap if they were not treated. Treatment begins during the early days or weeks of infancy with a carefully managed, phenylalanine-restricted diet to prevent intellectual impairment.
For more insights into Disease, causes, and treatment, reach out @ Phenylketonuria Treatment Landscape
Phenylketonuria Epidemiology Segmentation
The Phenylketonuria Market report proffers epidemiological analysis for the study period 2017-30 in the 7MM segmented into:
- Phenylketonuria Prevalence
- Age-Specific Phenylketonuria Prevalence
- Gender-Specific Phenylketonuria Prevalence
- Diagnosed and Treatable Cases of Phenylketonuria
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There is currently no cure for phenylketonuria, however, the most common treatment is a dietary restriction of phenylalanine to the bare minimum required for proper growth, supplemented with specially tailored medical meals.
For many years, the only treatment for Phenylketonuria was to follow a strict low-phenylalanine diet for the rest of one’s life. As a result, the primary line of treatment for Phenylketonuria is dietary therapy, which begins soon after diagnosis and consists of a low Phe diet supplemented with amino acid formulations.
The goal of Phenylketonuria treatment is to keep plasma phenylalanine levels between 120 and 360 mol/L (2–6 mg/dL), which can be accomplished with a properly designed and supervised diet. Because phenylalanine is an important amino acid, it should be limited in the child’s diet. A well-balanced diet can help people avoid intellectual handicaps, as well as neurological, behavioral, and dermatological issues.
Pharmaceutical companies’ extensive research and development activities, already approved therapies, and expanding prevalence will all contribute to the growth of the Phenylketonuria market.
Phenylketonuria Pipeline Therapies and Key Companies
- SYNB1618: Synlogic
- CNSA-001: Censa Pharmaceuticals
- HMI-102: Homology Medicines
- BMN 307: Biomarin Pharmaceuticals
For more information, visit Phenylketonuria Market Analysis, Patient Pool and Emerging Therapies
Phenylketonuria Market Drivers
- Rising prevalent population
- Technological advancements
- Emerging therapies
- Extensive R&D
Scope of the Report
- 11 Years Forecast
- 7MM Coverage
- Descriptive overview of Phenylketonuria, covering causes, signs and symptoms, pathophysiology, diagnosis, and currently available therapies
- Comprehensive insight into Phenylketonuria epidemiology and treatment in the 7MM
- Phenylketonuria Current and emerging therapies
- Phenylketonuria market drivers and barriers
Key Questions Answered in the Report
- What was the Phenylketonuria market share (%) distribution in 2017 and how it would appear in 2030?
- What would be the total Phenylketonuria market size and market size by therapies across the 7MM throughout the forecasted period (2017-2030)?
- What are the key findings from the 7MM market, and which country will have the largest Phenylketonuria market size during the forecasted period 2017 to 2030?
- At what CAGR, the Phenylketonuria market expected to grow by 7MM during the forecast period (2017-2030)?
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Table of Contents
|3.||Phenylketonuria Competitive Intelligence Analysis|
|4.||Phenylketonuria Market Overview at a Glance|
|5.||Phenylketonuria Executive Summary|
|6.||Phenylketonuria Epidemiology and Market Methodology|
|7.||Phenylketonuria Epidemiology and Patient Population|
|8.||Phenylketonuria Patient Journey|
|9.||Treatment Algorithm, Current Treatment, and Medical Practices|
|10.||Key Endpoints in Phenylketonuria Clinical Trials|
|11.||Phenylketonuria Marketed Therapies|
|12.||Phenylketonuria Emerging Therapies|
|13.||Phenylketonuria: 7 Major Market Analysis|
|15.||Phenylketonuria Access and Reimbursement Overview|
|18.||Phenylketonuria Market Drivers|
|19.||Phenylketonuria Market Barriers|
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