41 results on '"Saito, K."'
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2. VP.56a Real-world assessment of onasemnogene abeparvovec treatment in patients with spinal muscular atrophy: RESTORE/post-marketing surveillance in Japan
3. P.114 SUNFISH parts 1 and 2: 3-year efficacy and safety of risdiplam in types 2 and 3 spinal muscular atrophy (SMA)
4. P.104 Treatments and outcomes for patients with spinal muscular atrophy (SMA) type 2: Findings from RESTORE registry
5. P.105 Safety and effectiveness of onasemnogene abeparvovec (OA) alone or with other disease-modifying therapies (DMTs): Findings from RESTORE
6. SMA - TREATMENT
7. SMA - TREATMENT
8. SMA CLINICAL DATA
9. SMA - TREATMENT
10. SMA – THERAPY
11. SMA: REGISTRIES, BIOMARKERS & OUTCOME MEASURES
12. SMA – THERAPY
13. SMA – THERAPY
14. SMA THERAPIES I
15. SMA - TREATMENT: EP.290 Newborn screening (NBS) for spinal muscular atrophy (SMA) in the United States (US): Early findings from the RESTORE registry
16. SMA - TREATMENT: EP.289 Routine practices in use of onasemnogene abeparvovec (OA) in older patients with spinal muscular atrophy (SMA): Early findings from RESTORE
17. SMA - TREATMENT: EP.291 Real-world treatment patterns and outcomes in patients with spinal muscular atrophy (SMA): Updated findings from the RESTORE Registry
18. SMA - TREATMENT: EP.284 SUNFISH Part 2: 24-month efficacy and safety of risdiplam in patients with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA)
19. SMA - TREATMENT: EP.274 Onasemnogene Abeparvovec Gene Therapy in Presymptomatic spinal muscular atrophy (SMA): SPR1NT study update in children with 3 Copies of SMN2
20. SMA CLINICAL DATA: EP.249 ANCHOVY: A retrospective cohort study of the natural history of type 1 spinal muscular atrophy (SMA) using medical record data
21. The gross motor function measure is valid for Fukuyama congenital muscular dystrophy
22. A novel evaluation method of survival motor neuron protein as a biomarker of spinal muscular atrophy
23. The gross motor function measure is a valid scale for assessing Fukuyama congenital muscular dystrophy
24. Correlation between crural muscle fatty change and maximum motor function in Fukuyama congenital muscular dystrophy patients
25. G.P.40
26. G.P.318
27. G.P.317
28. G.P.316
29. P.8.3 Chronic respiratory failure in patients with Fukuyama congenital muscular dystrophy
30. G.P.269 - Correlation between crural muscle fatty change and maximum motor function in Fukuyama congenital muscular dystrophy patients
31. G.P.271 - The gross motor function measure is a valid scale for assessing Fukuyama congenital muscular dystrophy
32. G.P.24 - A novel evaluation method of survival motor neuron protein as a biomarker of spinal muscular atrophy
33. G.P.317: Renal dysfunction in patients with Fukuyama congenital muscular dystrophy
34. G.P.318: Natural history of motor function in patients with Fukuyama congenital muscular dystrophy
35. G.P.316: Effectiveness of levetiracetam for seizures in patients with Fukuyama congenital muscular dystrophy
36. G.P.40: Skeletal muscle MRI in an X-linked myotubular myopathy patient who became ambulatory
37. Merosin-positive congenital muscular dystrophy with mental retardation, microcephaly and central nervous system abnormalities unlinked to the Fukuyama muscular dystrophy and muscular–eye–brain loci: report of three siblings
38. Congenital muscular dystrophy with central and peripheral nervous system involvement in a Belgian patient
39. P.8.3 Chronic respiratory failure in patients with Fukuyama congenital muscular dystrophy.
40. SMA THERAPIES I: P.170Interim report on the safety and efficacy of longer-term treatment with nusinersen in infantile-onset spinal muscular atrophy: results from the SHINE study.
41. P.432 - The gross motor function measure is valid for Fukuyama congenital muscular dystrophy.
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