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

Rare Disease

spinal muscular atrophy, Ryukyuan type

MONDO:0010057

Also known as:Ryukyuan muscular atrophyspinal muscular atrophy Ryukyuan type

Overview

GeneticsAutosomal recessive

Treatments3 FDA approved, incl. 1 gene therapy

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: Kyphoscoliosis, Pes cavus, Fasciculations, and Spinal muscular atrophy and 1 more.

Organ System	Phenotype Count	Example Features
Muscles	2	Fasciculations, Spinal muscular atrophy
Bones and joints

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, Ryukyuan type, 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

Data assembled from 4 of 12 sources · Last updated Sep 20, 2026, 3:07 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

OMIM

Online Mendelian Inheritance in Man

OMIM:271200

GARD

Genetic and Rare Diseases Info Center

GARD:9646

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, Ryukyuan type?
→Should my partner and I meet with a genetic counselor to discuss carrier status and family planning?
→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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Kisho

spinal muscular atrophy, Ryukyuan type

Overview

Clinical Presentation

Treatment

Approved Therapies

Medical References

External Resources

OMIM

GARD

Questions for Your Care Team

Questions for Your Care Team

Explore clinical features

Clinical Trials

spinal muscular atrophy, Ryukyuan type

Overview

Clinical Presentation

Treatment

Approved Therapies

Medical References

External Resources

OMIM

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Explore clinical features

Clinical Trials