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Rhabdomyosarcoma is described in the packet as a rare, aggressive malignant mesenchymal neoplasm arising from skeletal muscle. The packet states that it usually occurs in children and young adults, that only a small percentage of tumors arise in the skeletal muscle of the extremities, and that the majority arise in other anatomical sites. The packet's onset categories are juvenile and childhood. Prevalence is not certified in this packet. The packet lists numerous subtypes, including embryonal, alveolar, spindle cell, and pleomorphic rhabdomyosarcoma, rhabdomyosarcoma with mixed embryonal and alveolar features, and many site-defined forms such as orbit, vaginal, and testis rhabdomyosarcoma.
The packet does not include phenotype annotations, characteristic findings, or a clinical description for rhabdomyosarcoma, so symptoms are not certified in this packet.
The packet lists no associated genes and no inheritance pattern for rhabdomyosarcoma, and it provides no molecular mechanism. The causes of the condition are therefore not certified in this packet.
The packet does not supply diagnostic criteria, testing methods, or newborn screening information for rhabdomyosarcoma. Diagnosis is therefore not certified in this packet.
The packet lists no FDA-approved treatments and no foundational therapies for rhabdomyosarcoma, so treatment coverage is not certified in this packet. The packet's orphan drug records include one entry, padnarsertib, with a DESIGNATED status for treatment of rhabdomyosarcoma; orphan designation is not approval.
90 trials found
The packet does not include natural history, survival, or outcome information for rhabdomyosarcoma. Prognosis is not certified in this packet. The packet describes the tumor as aggressive.
The packet reports 86 active clinical trials for rhabdomyosarcoma on ClinicalTrials.gov, with intervention types including drug therapy, procedural interventions, biologic therapy, and other interventions, and the presence of gene therapy trials. Detailed records include NCT06865664, a Phase 1 study of FGFR4 chimeric antigen receptor T cells in children and young adults with recurrent or refractory rhabdomyosarcoma, sponsored by the National Cancer Institute (recruiting; 2025-09-22 to 2029-04-01); and NCT03618381, a Phase 1 study of EGFR806 CAR T cells for recurrent or refractory solid tumors sponsored by Seattle Children's Hospital (active, not recruiting). Several listed records are Pediatric MATCH or pediatric solid tumor studies that are not specific to rhabdomyosarcoma. Titles alone do not establish the intervention studied. The packet's research landscape digest classifies 662 publications, with case reports and case series the dominant type.
Data assembled from 4 of 12 sources · Last updated Nov 3, 2026, 3:00 PM UTC
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Genetic and Rare Diseases Info Center
AI-curated news mentioning rhabdomyosarcoma
Updated Aug 17, 2026
A new overview on pediatric rhabdomyosarcoma in the head and neck has been published, providing insights into the disease's characteristics and treatment approaches. This research contributes to the understanding of this rare cancer in children.
A case report highlights the successful use of sequential multimodal therapy for treating lingual rhabdomyosarcoma in a young adult. This approach may provide insights into effective treatment strategies for this rare cancer.
Research indicates that high levels of dynein negatively affect mitochondrial distribution and differentiation in rhabdomyosarcoma cells. This study provides insights into the cellular mechanisms that could influence treatment strategies for this rare cancer.