Works matching IS 26330040 AND DT 2023 AND VI 4
Results: 17
The Rare Diseases Clinical Research Network: a model for clinical trial readiness.
- Published in:
- Therapeutic Advances in Rare Disease, 2023, v. 4, p. 1, doi. 10.1177/26330040231219272
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- Publication type:
- Article
The pharmacological treatment of granulomatosis with polyangiitis: a review of clinical trials registered in clinicaltrials.gov and the International Clinical Trials Registry Platform.
- Published in:
- Therapeutic Advances in Rare Disease, 2023, v. 4, p. 1, doi. 10.1177/26330040231213888
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- Publication type:
- Article
Study protocol of the HD-MED study aiming to personalize drug treatment in Huntington's disease: a longitudinal, observational study to assess medication use and efficacy in relation to pharmacogenetics.
- Published in:
- Therapeutic Advances in Rare Disease, 2023, v. 4, p. 1, doi. 10.1177/26330040231204643
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- Publication type:
- Article
26th Annual Symposium of the United Mitochondrial Disease Foundation -Mitochondrial Medicine 2023 – Abstracts.
- Published in:
- 2023
- Publication type:
- Abstract
Case report on the use of canakinumab for treatment of recurrent fevers and proteinuria in refractory systemic lupus erythematosus.
- Published in:
- Therapeutic Advances in Rare Disease, 2023, v. 4, p. 1, doi. 10.1177/26330040231191141
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- Publication type:
- Article
A multidisciplinary approach to treating a unique case of recurrent metastatic thymic carcinoma: case report.
- Published in:
- Therapeutic Advances in Rare Disease, 2023, v. 4, p. 1, doi. 10.1177/26330040231190661
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- Publication type:
- Article
The IRDiRC Chrysalis Task Force: making rare disease research attractive to companies.
- Published in:
- Therapeutic Advances in Rare Disease, 2023, v. 4, p. 1, doi. 10.1177/26330040231188979
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- Publication type:
- Article
Surgical resection therapy of a rare presentation of persistent Mullerian duct syndrome: a case review.
- Published in:
- Therapeutic Advances in Rare Disease, 2023, v. 4, p. 1, doi. 10.1177/26330040231184484
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- Publication type:
- Article
Toward representative genomic research: the children's rare disease cohorts experience.
- Published in:
- Therapeutic Advances in Rare Disease, 2023, v. 4, p. 1, doi. 10.1177/26330040231181406
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- Publication type:
- Article
Enhancing diversity, equity, inclusion, and accessibility in eosinophilic gastrointestinal disease research: the consortium for eosinophilic gastrointestinal disease researchers' journey.
- Published in:
- Therapeutic Advances in Rare Disease, 2023, v. 4, p. 1, doi. 10.1177/26330040231180895
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- Publication type:
- Article
Bulbar conjunctival plexiform schwannoma in a 5-year-old patient; expect the unexpected!
- Published in:
- Therapeutic Advances in Rare Disease, 2023, v. 4, p. 1, doi. 10.1177/26330040231178321
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- Publication type:
- Article
Emerging roles and opportunities for rare disease patient advocacy groups.
- Published in:
- Therapeutic Advances in Rare Disease, 2023, v. 4, p. 1, doi. 10.1177/26330040231164425
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- Publication type:
- Article
A novel case of homozygous PAX1 mutation associated with hypoparathyroidism.
- Published in:
- Therapeutic Advances in Rare Disease, 2023, v. 4, p. 1, doi. 10.1177/26330040231158776
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- Publication type:
- Article
Successful combined umbilical cord blood and bone marrow transplantation from an HLA-matched sibling for MPS VI: a case report.
- Published in:
- Therapeutic Advances in Rare Disease, 2023, v. 4, p. 1, doi. 10.1177/26330040231154283
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- Publication type:
- Article
Papilledema and retinopathy lead to diagnosis of IgA nephropathy: a case report.
- Published in:
- Therapeutic Advances in Rare Disease, 2023, v. 4, p. 1, doi. 10.1177/26330040231152957
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- Publication type:
- Article
Novel insights into the phenotype and long-term D-gal treatment in PGM1-CDG: a case series.
- Published in:
- Therapeutic Advances in Rare Disease, 2023, v. 4, p. 1, doi. 10.1177/26330040221150269
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- Publication type:
- Article
Left-sided valvular heart disease and retinopathy in a 38-year-old woman with attenuated mucopolysaccharidosis: a case report.
- Published in:
- Therapeutic Advances in Rare Disease, 2023, v. 4, p. 1, doi. 10.1177/26330040221145945
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- Publication type:
- Article