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multiple system atrophy (MSA) - Clinical Profile, Treatments & Trials | Kisho | Kisho

Rare Disease

multiple system atrophy

MONDO:0007803

Also known as:MSAShy-Drager syndromemultisystem atrophy

Find Clinical TrialsTrials

Overview

Multiple system atrophy (MSA) is a neurodegenerative disorder characterized by autonomic failure (cardiovascular and/or urinary), parkinsonism, cerebellar impairment and corticospinal signs with a median survival of 6-9 years.

Treatments1 FDA approved

Clinical Trials97 registered · 44 recruiting

Patient Orgs3 organizations · Defeat Multiple System Atrophy Alliance, Mission MSA + 1 more

Clinical Presentation

Built from Human Phenotype Ontology (HPO) phenotype annotations and GeneReviews clinical descriptions. Features represent findings documented across known cases — not an exhaustive list of possible symptoms.

Features include common findings: Gaze-evoked nystagmus, Dysarthria, Parkinsonism, and Constipation and others.

Organ System	Phenotype Count	Example Features
Brain and nerves	15	Dysarthria, Parkinsonism, Muscle stiffness (rigidity)
Eyes

Diagnosis

Biomarker and diagnostic research for multiple system atrophy has been reported in the published literature.

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

1 FDA-approved treatment is available for multiple system atrophy, including DROXIDOPA (NORTHERA, approved 2014). An additional 7 compounds hold orphan drug designation.

Approved Therapies

Brand Name	Generic Name	Mechanism	Approved	Market Status

Prognosis

Phenotype severity distribution: 27 common features.

Estimated prevalence: 1-9 in 100,000 (Uncommon).

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

97 clinical trials registered, 44 recruiting. Interventions under study include other interventions, drug therapy, medical devices, and biologic therapy. Pipeline includes 1 PHASE4, 3 PHASE3, 13 PHASE2. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT06920134](https://clinicaltrials.gov/study/NCT06920134)

Data assembled from 6 of 12 sources · Last updated Sep 19, 2026, 3:00 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Patient Support

Patient Advocacy Groups

View all PAGs →

Defeat Multiple System Atrophy AlliancePrimary Focus

USWebsite ↗

Mission MSAPrimary Focus

USWebsite ↗

CurePSP

USWebsite ↗

Patient Advocacy Groups (PAGs) provide support, resources, and community for patients and caregivers.

Are you a patient organization not listed here? Claim this page to be listed and embed this content on your site — free.

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:102

GARD

Genetic and Rare Diseases Info Center

GARD:7079

Questions for Your Care Team

→What are the benefits and potential side effects of NORTHERA for managing multiple system atrophy?
→Are any of the 97 active clinical trials a good fit for my situation?
→Are there specialists or centers of excellence for this condition in my area?
→How can I connect with Defeat Multiple System Atrophy Alliance and other patient organizations for support?

← Back to All Diseases

Brand Name	Generic Name	Sponsor	Designated	Exclusivity End	Designation Status
exidavnemab	exidavnemab	BioArctic AB	2025	—	Designated
human recombinant, monoclonal antibody (mAb) of the IgG1 isotype against alpha-synuclein	human recombinant, monoclonal antibody (mAb) of the IgG1 isotype against alpha-synuclein	Lundbeck Pharmaceuticals LLC	2024	—	Designated
N-(4-Methyl-3-(4-(5-(4-methylisoxazol-5-yl)pyridine-3-yl)pyrimidin-2-yl)amino)phenyl)-4-((4-methylpiperazin-1-yl)methyl)benzamide succinate	N-(4-Methyl-3-(4-(5-(4-methylisoxazol-5-yl)pyridine-3-yl)pyrimidin-2-yl)amino)phenyl)-4-((4-methylpiperazin-1-yl)methyl)benzamide succinate	ABLi Therapeutics, Inc.	2023	—	Designated
3-(1,3-benzodioxol-5-yl)-5-(3-bromophenyl)-1H-pyrazole	3-(1,3-benzodioxol-5-yl)-5-(3-bromophenyl)-1H-pyrazole	Teva Branded Pharmaceutical Products R&D, Inc.	2022	—	Designated
2-[[(4-Methoxy-3,5-dimethyl-2-pyridinyl)methyl]sulfinyl]-1H-benzimidazol-5-ol or 5-O-desmethyl-omeprazole	2-[[(4-Methoxy-3,5-dimethyl-2-pyridinyl)methyl]sulfinyl]-1H-benzimidazol-5-ol or 5-O-desmethyl-omeprazole	Yoda Therapeutics Inc.	2022	—	Designated
verdiperstat	verdiperstat	Biohaven Pharmaceuticals, Inc.	2019	—	Designated
5,7-Dichloro-2-((ethylamino)methyl)-8-hydroxy-3-methylquinazolin-4(3H)-one mesylate	5,7-Dichloro-2-((ethylamino)methyl)-8-hydroxy-3-methylquinazolin-4(3H)-one mesylate	Alterity Therapeutics, Limited	2019	—	Designated

Research Type	Count	% of Total
Laboratory research	97	26%
Testing and diagnosis research	95	25%
Research summaries	82	22%
Disease patterns and progression	51	14%
Patient case studies	28	7%
Clinical study results	17	5%
Other research	4	1%
New treatment approaches	3	1%

Kisho

multiple system atrophy

Overview

Clinical Presentation

Diagnosis

Treatment

Approved Therapies

Prognosis

Research Landscape

Patient Support

Patient Advocacy Groups

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Explore clinical features

Orphan Drug Designations

Clinical Trials

Recent Publications

Latest News & Developments

Plasma neurofilament light for early differentiation of multiple system atrophy from Parkinson disease.

[Multiple system atrophy].

Magnetic resonance imaging markers predict disease progression in early-stage multiple system atrophy: a 2-year prospective cohort study.

Lundbeck completes patient randomisation early in global MSA trial

multiple system atrophy

Overview

Clinical Presentation

Diagnosis

Treatment

Approved Therapies

Prognosis

Research Landscape

Patient Support

Patient Advocacy Groups

Medical References

External Resources

Orphanet

GARD

Subtypes

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Explore clinical features

Orphan Drug Designations

Clinical Trials

Recent Publications

Latest News & Developments

Plasma neurofilament light for early differentiation of multiple system atrophy from Parkinson disease.

[Multiple system atrophy].

Magnetic resonance imaging markers predict disease progression in early-stage multiple system atrophy: a 2-year prospective cohort study.

Lundbeck completes patient randomisation early in global MSA trial