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Muscular Dystrophy Association (MDA), 2025

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Survival analyses in patients with thymidine kinase 2 deficiency aged ≤12 years at symptom onset who received pyrimidine nucleos(t)ide therapy
Hirano Michio, Garone Caterina, Haas Richard, et al.
Poster
Survival and functional outcomes in patients with thymidine kinase 2 deficiency aged >12 years at symptom onset who received pyrimidine nucleos(t)ides
Scaglia Fernando, Hirano Michio, Garone Caterina, et al.
Poster
Functional outcomes in patients with thymidine kinase 2 deficiency aged ≤12 years at symptom onset who received pyrimidine nucleos(t)ide therapy
Garone Caterina, Hirano Michio, Haas Richard, et al.
Poster
Burden and impact of caring for those with thymidine kinase 2 deficiency (TK2d): results from the Assessment of TK2d Patient Perspectives (ATP) study
Yeske Philip, Balcells Cristy, Hareendran Asha, et al.
Poster
Patients’ lived experience of thymidine kinase 2 deficiency (TK2d): results from the Assessment of TK2d Patient Perspectives (ATP) study
Yeske Philip, Balcells Cristy, Hareendran Asha, et al.
Poster
Response rates with zilucoplan in generalized myasthenia gravis: 120-week interim analysis of RAISE-XT
James F. Howard Jr., Freimer Miriam, Genge Angela, et al.
Poster
Response over time with zilucoplan in generalized myasthenia gravis: Post hoc analysis of RAISE-XT 60-week follow up
Hewamadduma Channa, Bresch Saskia, Freimer Miriam, et al.
Poster
Ocular symptoms in patients with generalized myasthenia gravis receiving rozanolixizumab: Post hoc analysis of MycarinG
Habib A. Ali, Pascuzzi M. Robert, Vissing John, et al.
Poster
Safety and efficacy of chronic weekly rozanolixizumab treatment in patients with generalized myasthenia gravis (MG0004)
Bril Vera, Drużdż Artur, Grosskreutz Julian, et al.
Poster
The Diagnostic Dilemma: Genetic Testing vs. Muscle Biopsy in Mitochondrial Myopathies
Dr. Michio Hirano
Medical Symposium