25 results on '"Fischer, D"'
Search Results
2. SMA - TREATMENT
3. SMA - TREATMENT
4. SMA – THERAPY
5. P.363JEWELFISH: safety and pharmacodynamic data in patients with spinal muscular atrophy (SMA) receiving treatment with risdiplam (RG7916) that have previously been treated with nusinersen
6. DMD CLINICAL AND BIOMARKERS
7. P.190Feasibility, reliability and convergent validity for digital biomarkers captured via a smartphone application (app) to assess motor behaviors in individuals with spinal muscular atrophy (SMA) in the JEWELFISH trial
8. SMA THERAPIES II AND BIOMARKERS
9. SMA THERAPIES II AND BIOMARKERS
10. DMD CLINICAL THERAPIES I
11. SMA - TREATMENT: EP.280 Pooled safety data from the risdiplam clinical trial development program
12. SMA - TREATMENT: EP.279 JEWELFISH: Safety, pharmacodynamic and exploratory efficacy data in non-naïve patients with spinal muscular atrophy (SMA) receiving risdiplam
13. Repurposing tamoxifen for severe myopathies: from preclinical evaluation in animal models to clinical trials in patients
14. A series of case reports from JEWELFISH, an open-label study to investigate the safety, tolerability, and pharmacokinetics/ pharmacodynamics of RG7916 in adult and pediatric patients with spinal muscular atrophy who previously participated in a study with another SMN2-targeting therapy
15. Towards objective and reproducible measures of thigh muscle fat fraction in patients with Duchenne Muscular Dystrophy
16. P.376 - A series of case reports from JEWELFISH, an open-label study to investigate the safety, tolerability, and pharmacokinetics/ pharmacodynamics of RG7916 in adult and pediatric patients with spinal muscular atrophy who previously participated in a study with another SMN2-targeting therapy
17. D06 - Towards objective and reproducible measures of thigh muscle fat fraction in patients with Duchenne Muscular Dystrophy
18. M.P.5.04 Evaluation of in-phase and out-of-phase and FISP MRI to quantify muscle fat content in OPMD
19. M.P.5.05 Whole-body muscle MRI in collagen type VI-related myopathies (Ullrich CMD and Bethlem myopathy)
20. G.O.7 A homozygous desmin deletion causes an Emery-Dreifuss like recessive myopathy with desmin depletion
21. G.O.1 The phenotype of myofibrillar myopathy associated with p.W2710X mutation in filamin C: A study of 31 German patients
22. C.P.4.02 Muscle imaging differentiates primary desminopathies from myofibrillar myopathies
23. C.P.4.01 Clinical and cardiac MRI findings in primary desminopathies
24. P.411 - Repurposing tamoxifen for severe myopathies: from preclinical evaluation in animal models to clinical trials in patients.
25. G.P.8 08 Identification of a desmin gene mutation in scapuloperoneal syndrome type Kaeser
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