
Maple Syrup Urine Disease- Pipeline Insight, 2025
Description
DelveInsight’s, “Maple Syrup Urine Disease- Pipeline Insight, 2025” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Maple Syrup Urine Disease 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.
Geography Covered
Maple Syrup Urine Disease: Overview
Maple syrup urine disease (MSUD) is a rare genetic metabolic disorder called an inborn error of metabolism (IEM). For people with MSUD, the body does not have enough of a properly working enzyme needed to break down certain amino acids (building blocks of protein).This enzyme is called the branched chain alpha ketoacid dehydrogenase (BCKD) complex. MSUD is caused by mutations in the genes involved in making the BCKD complex, which breaks down the amino acids leucine, isoleucine, and valine (also called branched-chain amino acids, BCAA). These amino acids are found in protein-rich foods. The amino acids and their by-products build up in the bodies of people with MSUD, and can result in damage to the brain and other organs. MSUD is inherited in an autosomal recessive pattern, which means that both parents have one defective copy of the gene, which is passed on to the affected child. MSUD occurs in approximately 1 in 185,000 infants, but is more common in Old Order Mennonites, where it has a frequency of 1 in 380. It is equally likely to affect males and females. MSUD is sometimes subdivided into 4 types: classic, intermediate, intermittent and thiamine-responsive. Diagnosis of MSUD is based on symptoms and biochemical testing of blood and urine. It is confirmed with molecular genetic testing for mutations in the genes that encode BCKD. Tests may also be done to measure enzyme activity but this testing is becoming less readily available. MSUD can also be detected using newborn screening, which is now being performed in the U.S. and other developed countries. There are two main aspects to managing all types of MSUD: chronic treatment with a special diet and acute treatment of a metabolic crisis (where the stress of fasting, dehydration, and/or a serious illness triggers severe symptoms) when it occurs.
“Maple Syrup Urine Disease- Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Maple Syrup Urine Disease pipeline landscape is provided which includes the disease overview and Maple Syrup Urine Disease treatment guidelines. The assessment part of the report embraces, in depth Maple Syrup Urine Disease commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Maple Syrup Urine Disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
This segment of the Maple Syrup Urine Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Maple Syrup Urine Disease Emerging Drugs
Further product details are provided in the report……..
Maple Syrup Urine Disease: Therapeutic Assessment
This segment of the report provides insights about the different Maple Syrup Urine Disease drugs segregated based on following parameters that define the scope of the report, such as:
Maple Syrup Urine Disease: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Maple Syrup Urine Disease therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Maple Syrup Urine Disease drugs.
Maple Syrup Urine Disease Report Insights
Current Treatment Scenario and Emerging Therapies:
Geography Covered
- Global coverage
Maple Syrup Urine Disease: Overview
Maple syrup urine disease (MSUD) is a rare genetic metabolic disorder called an inborn error of metabolism (IEM). For people with MSUD, the body does not have enough of a properly working enzyme needed to break down certain amino acids (building blocks of protein).This enzyme is called the branched chain alpha ketoacid dehydrogenase (BCKD) complex. MSUD is caused by mutations in the genes involved in making the BCKD complex, which breaks down the amino acids leucine, isoleucine, and valine (also called branched-chain amino acids, BCAA). These amino acids are found in protein-rich foods. The amino acids and their by-products build up in the bodies of people with MSUD, and can result in damage to the brain and other organs. MSUD is inherited in an autosomal recessive pattern, which means that both parents have one defective copy of the gene, which is passed on to the affected child. MSUD occurs in approximately 1 in 185,000 infants, but is more common in Old Order Mennonites, where it has a frequency of 1 in 380. It is equally likely to affect males and females. MSUD is sometimes subdivided into 4 types: classic, intermediate, intermittent and thiamine-responsive. Diagnosis of MSUD is based on symptoms and biochemical testing of blood and urine. It is confirmed with molecular genetic testing for mutations in the genes that encode BCKD. Tests may also be done to measure enzyme activity but this testing is becoming less readily available. MSUD can also be detected using newborn screening, which is now being performed in the U.S. and other developed countries. There are two main aspects to managing all types of MSUD: chronic treatment with a special diet and acute treatment of a metabolic crisis (where the stress of fasting, dehydration, and/or a serious illness triggers severe symptoms) when it occurs.
“Maple Syrup Urine Disease- Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Maple Syrup Urine Disease pipeline landscape is provided which includes the disease overview and Maple Syrup Urine Disease treatment guidelines. The assessment part of the report embraces, in depth Maple Syrup Urine Disease commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Maple Syrup Urine Disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
- The companies and academics are working to assess challenges and seek opportunities that could influence Maple Syrup Urine Disease R&D. The therapies under development are focused on novel approaches to treat/improve Maple Syrup Urine Disease.
This segment of the Maple Syrup Urine Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Maple Syrup Urine Disease Emerging Drugs
- ACER 001: Acer Therapeutics
Further product details are provided in the report……..
Maple Syrup Urine Disease: Therapeutic Assessment
This segment of the report provides insights about the different Maple Syrup Urine Disease drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Maple Syrup Urine Disease
- Phases
- Late stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage product (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
- Intra-articular
- Intraocular
- Intrathecal
- Intravenous
- Ophthalmic
- Oral
- Parenteral
- Subcutaneous
- Topical
- Transdermal
- Molecule Type
- Oligonucleotide
- Peptide
- Small molecule
- Product Type
Maple Syrup Urine Disease: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Maple Syrup Urine Disease therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Maple Syrup Urine Disease drugs.
Maple Syrup Urine Disease Report Insights
- Maple Syrup Urine Disease Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Maple Syrup Urine Disease drugs?
- How many Maple Syrup Urine Disease drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Maple Syrup Urine Disease?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Maple Syrup Urine Disease therapeutics?
- What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Maple Syrup Urine Disease and their status?
- What are the key designations that have been granted to the emerging drugs?
- Acer Therapeutics
- ASC Therapeutics
- APR Applied Pharma Research
- LyGenesis
Key Products- ACER 001
- Gene therapy
- APR OM33
- REC 0545
Table of Contents
60 Pages
- Introduction
- Executive Summary
- Maple Syrup Urine Disease: Overview
- Causes
- Mechanism of Action
- Signs and Symptoms
- Diagnosis
- Disease Management
- Pipeline Therapeutics
- Comparative Analysis
- Therapeutic Assessment
- Assessment by Product Type
- Assessment by Stage and Product Type
- Assessment by Route of Administration
- Assessment by Stage and Route of Administration
- Assessment by Molecule Type
- Assessment by Stage and Molecule Type
- Maple Syrup Urine Disease– DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Comparative Analysis
- Drug name: Company name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- Comparative Analysis
- ACER 001: Acer Therapeutics
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- Comparative Analysis
- Drug name: Company name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Comparative Analysis
- Drug name: Company name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Inactive Products
- Comparative Analysis
- Maple Syrup Urine Disease Key Companies
- Maple Syrup Urine Disease Key Products
- Maple Syrup Urine Disease - Unmet Needs
- Maple Syrup Urine Disease - Market Drivers and Barriers
- Maple Syrup Urine Disease - Future Perspectives and Conclusion
- Maple Syrup Urine Disease Analyst Views
- Maple Syrup Urine Disease Key Companies
- Appendix
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