Kisho

Kisho is an information platform, not a medical provider. Nothing on this site constitutes medical advice, diagnosis, or treatment recommendations. All content is aggregated from publicly available sources (including ClinicalTrials.gov, PubMed, FDA.gov, and Orphanet) and is provided for informational purposes only. Clinical trial eligibility, treatment decisions, and any health-related actions should always be discussed with a qualified healthcare professional. Kisho does not endorse any specific therapy, organization, or clinical trial. Terms of use · Privacy policy

muscular dystrophy - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

muscular dystrophy

MONDO:0020121

Find Clinical TrialsTrials

Overview

Muscular dystrophy (MD) refers to a group of more than 30 genetic diseases characterized by progressive weakness and degeneration of the skeletal muscles that control movement. Some forms of MD are seen in newborns, infants or children, while others have late-onset and may not appear until middle age or later. The disorders differ in terms of the distribution and extent of muscle weakness (some forms of MD also affect cardiac muscle), age of onset, rate of progression, and pattern of inheritance. The prognosis for people with MD varies according to the type and progression of the disorder. There is no specific treatment to stop or reverse any form of MD. Treatment is supportive and may include physical therapy, respiratory therapy, speech therapy, orthopedic appliances used for support, corrective orthopedic surgery, and medicationsincluding corticosteroids, anticonvulsants (seizure medications), immunosuppressants, and antibiotics. Some individuals may need assisted ventilation to treat respiratory muscle weaknessor a pacemaker for cardiac (heart)abnormalities.

Clinical Trials210 registered · 93 recruiting

Patient Orgs4 organizations · Muscular Dystrophy Association, Muscular Dystrophy Association of New Zealand, Inc. + 2 more

Diagnosis

Biomarker and diagnostic research for muscular dystrophy has been reported in the published literature.

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.

210 clinical trials registered, 93 recruiting. Interventions under study include other interventions, drug therapy, gene therapy, and medical devices. Pipeline includes 4 PHASE4, 16 PHASE3, 28 PHASE2. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT07435129](https://clinicaltrials.gov/study/NCT07435129)

Data assembled from 4 of 12 sources · Last updated Sep 18, 2026, 6:00 AM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Patient Support

Patient Advocacy Groups

View all PAGs →

Muscular Dystrophy AssociationPrimary Focus

USWebsite ↗

Muscular Dystrophy Association of New Zealand, Inc.Primary Focus

USWebsite ↗

Muscular Dystrophy CanadaPrimary Focus

USWebsite ↗

Muscular Dystrophy IrelandPrimary Focus

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:98473

GARD

Genetic and Rare Diseases Info Center

GARD:7922

Questions for Your Care Team

→Are any of the 210 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 Muscular Dystrophy Association and other patient organizations for support?

← Back to All Diseases

Research Type	Count	% of Total
Laboratory research	94	29%
Research summaries	68	21%
New treatment approaches	56	17%
Disease patterns and progression	41	13%
Testing and diagnosis research	23	7%
Other research	16	5%
Clinical study results	15	5%
Patient case studies	9	3%

Kisho

muscular dystrophy

Overview

Diagnosis

Research Landscape

Patient Support

Patient Advocacy Groups

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Recent Publications

Latest News & Developments

After Novartis’ devastating M&A losses, long live the executive willing to try

Novo’s canned heart disease trials, Novartis’ clinical fails, first approval for Alexander disease

INPP5K-related muscular dystrophy caused by a novel synonymous splicing variant in a Chinese patient: a case report.

Novartis’ $12B Avidity buy pays dividends with Phase 1/2 muscular dystrophy win

Advanced Therapy Medicinal Products Market Size to Reach US$ 46.11 Billion by 2034, Growing at 14.79% CAGR During 2026-2034

muscular dystrophy

Overview

Diagnosis

Research Landscape

Patient Support

Patient Advocacy Groups

Medical References

External Resources

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Recent Publications

Latest News & Developments

After Novartis’ devastating M&A losses, long live the executive willing to try

Novo’s canned heart disease trials, Novartis’ clinical fails, first approval for Alexander disease

INPP5K-related muscular dystrophy caused by a novel synonymous splicing variant in a Chinese patient: a case report.

Novartis’ $12B Avidity buy pays dividends with Phase 1/2 muscular dystrophy win

Advanced Therapy Medicinal Products Market Size to Reach US$ 46.11 Billion by 2034, Growing at 14.79% CAGR During 2026-2034