Multiple System Atrophy (MSA)
Multiple system atrophy (MSA) is a rare, rapidly progressive neurodegenerative disorder. Protheragen leverages cutting-edge insights into the pathogenesis of MSA to pioneer targeted therapies and precision animal models, accelerating preclinical drug development. Our goal is to offer reliable and end-to-end support to streamline your therapeutic development journey.
Introduction to Multiple System Atrophy (MSA)
Multiple system atrophy (MSA) is an uncommon, quickly advancing neurodegenerative disease marked by glial cytoplasmic inclusions (GCIs) containing excess α-synuclein and widespread degeneration of nervous system tissues. Occurring at an annual rate of 0.6 to 3 new cases per 100,000 individuals, MSA usually results in marked disability and death 6 to 10 years after symptoms first appear. Clinically, MSA is classified into two principal phenotypes: the parkinsonian type (MSA-P), which comprises approximately 60 percent of cases, and the cerebellar type (MSA-C), which accounts for the remaining 40 percent.
Fig.1 Clinical signs for diagnostic assessment of multiple system atrophy (MSA) subtypes. (Goolla M, et al., 2023)
Pathogenesis of Multiple System Atrophy (MSA)
Multiple system atrophy (MSA) arises primarily when α-synuclein misfolds and aggregates inside oligodendrocytes, generating glial cytoplasmic inclusions (GCIs) that disrupt myelin sheaths, provoke neuroinflammation, and drive neurodegeneration. This process is aggravated by mitochondrial dysfunction and the prion-like spread of misfolded α-synuclein along central autonomic and motor pathways, culminating in the widespread, multisystem failures.
Fig.2 Clinical and pathological features of multiple system atrophy (MSA). (Kinoshita C, et al., 2022)
Therapeutic Development for Multiple System Atrophy (MSA)
| Drug Names | Mechanism of Action | Targets | NCT Number | Research Phase |
| Verdiperstat | Irreversible myeloperoxidase (MPO) inhibitor reducing oxidative stress and neuroinflammation | Myeloperoxidase (MPO) enzyme | NCT05086094 | Phase III |
| Safinamide | Dual MAO-B inhibitor and glutamate release modulator | MAO-B enzyme; voltage-gated sodium channels | NCT03753763 | Phase II/III |
| Lu AF82422 | Humanized monoclonal antibody targeting pathological α-synuclein conformers | Oligomeric α-synuclein in glial cytoplasmic inclusions (GCIs) | NCT06706622 | Phase I |
Disclaimer: Protheragen focuses on providing preclinical research services. This table is for information exchange purposes only. This table is not a treatment plan recommendation. For guidance on treatment options, please visit a regular hospital.
Our Services
Recognizing the complexity of diagnosing and treating multiple system atrophy (MSA), Protheragen is committed to building a team of experts to provide cutting-edge diagnostic and therapeutic development solutions. Our commitment lies in providing a variety of customized therapy development services to meet the diverse research needs of our customers. We also excel in generating precise disease models that are carefully engineered to replicate the unique features of MSA.
Therapeutic Development Services
By Mechanism of Action
Disease Model Development Services
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At Protheragen, our passion lies in empowering the next generation of groundbreaking therapies. We offer a full suite of preclinical research services, from pharmacodynamics (PD) and pharmacokinetic (PK) to toxicology, that can be tailored to your specific needs. If you are interested in our services, please feel free to contact usfor more details and quotation information of related services.
References
- Goolla M, Cheshire W P, Ross O A, et al. Diagnosing multiple system atrophy: current clinical guidance and emerging molecular biomarkers[J]. Frontiers in Neurology, 2023, 14: 1210220.
- Kinoshita C, Kubota N, Aoyama K. Glutathione depletion and microRNA dysregulation in multiple system atrophy: a review[J]. International Journal of Molecular Sciences, 2022, 23(23): 15076.