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spinal muscular atrophy type 0 - Clinical Profile, Treatments & Trials | Kisho | Kisho

Rare Disease

spinal muscular atrophy type 0

MONDO:0850170

Overview

A childhood spinal muscular atrophy that is evident before birth and characterized by diminished movement in the womb, joint deformities, extremely weak muscle tone and very weak respiratory muscles.

Treatments3 FDA approved, incl. 1 gene therapy

ResearchLimited published research (7 articles)

Diagnosis

Biomarker and diagnostic research for spinal muscular atrophy type 0 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.

3 FDA-approved treatments are available for spinal muscular atrophy type 0, including NUSINERSEN (SPINRAZA, approved 2016), onasemnogene abeparvovec-xioi (Zolgensma, approved 2019), and RISDIPLAM (EVRYSDI, approved 2020).

Approved Therapies

Brand Name	Generic Name	Mechanism	Approved	Market Status

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.

No clinical trials have been registered for spinal muscular atrophy type 0.

7 publications have been identified in PubMed for spinal muscular atrophy type 0. Research spans Case Report / Case Series (57%), Diagnostic / Biomarker (14%), and Review / Meta-Analysis (14%).

Recent Publications

Okubo Y (2026). [PMID: 41795276](https://pubmed.ncbi.nlm.nih.gov/41795276/). *Brain & development*. [Case Report / Case Series]
Codina-Solà M (2026). [PMID: 41531030](https://pubmed.ncbi.nlm.nih.gov/41531030/). *Prenatal diagnosis*. [Diagnostic / Biomarker]
Kimizu T (2025). [PMID: 40163981](https://pubmed.ncbi.nlm.nih.gov/40163981/). *Brain & development*. [Case Report / Case Series]
Uchio Y (2025). [PMID: 40511310](https://pubmed.ncbi.nlm.nih.gov/40511310/). *Journal of physical therapy science*. [Epidemiology / Natural History]
Stokes S (2025). [PMID: 40923389](https://pubmed.ncbi.nlm.nih.gov/40923389/). *Molecular genetics & genomic medicine*. [Review / Meta-Analysis]

Data assembled from 3 of 12 sources · Last updated Sep 19, 2026, 9:39 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

Questions for Your Care Team

→What are the eligibility criteria and monitoring requirements for Zolgensma?
→What are the benefits and potential side effects of SPINRAZA for managing spinal muscular atrophy type 0?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

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spinal muscular atrophy type 0

Overview

Diagnosis

Treatment

Approved Therapies

Research Landscape

Recent Publications

Medical References

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials