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2. P444 Ophthalmologic findings following intrathecal AAV9 mediated gene transfer for Giant Axonal Neuropathy

3. O16 New FDX2-loss of function phenotype presenting with blindness and myopathy with potential responsiveness to Co-enzyme Q10 analogs

4. P285 Bi-allelic variants in HMGCR cause limb girdle muscular dystrophy and further implicate the mevalonate pathway in muscle disease

5. P445 Electrophysiologic and histologic findings following intrathecal AAV9 mediated gene transfer for giant axonal neuropathy

7. O.10 First-in-human intrathecal gene transfer study for giant axonal neuropathy: Preliminary review of long-term efficacy and safety

8. O.06 Long term outcomes for X-Linked myotubular Myopathy (XLMTM) with gene replacement therapy, resamirigene bilparvovec: Preliminary results from ASPIRO

9. United Arab Emirates National Newborn Screening Programme:an evaluation 1998-2000

10. Improved Respiratory Outcomes for X-Linked Myotubular Myopathy (XLMTM) with Gene Replacement Therapy, Resamirigene Bilparvovec (ASPIRO): Preliminary Results from ASPIRO, a Phase 1/2/3 Study

11. ASPIRO Gene Therapy Trial in X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Efficacy and Safety Findings

12. OUTCOME MEASURES

13. COLLAGEN RELATED MUSCLE DISEASES

14. CLINICAL TRIAL HIGHLIGHTS

15. COLLAGEN RELATED MUSCLE DISEASES

17. HEREDITARY NEUROPATHIES & ALS

18. CONGENITAL MYOPATHIES 1 – NEMALINE

19. HEREDITARY NEUROPATHIES & ALS

20. An ultrafast system for signaling mechanical pain in human skin

21. Recessive mutations in muscle-specific isoforms of FXR1 cause congenital multi-minicore myopathy

23. DISTAL MYOPATHIES

24. CONGENITAL MUSCULAR DYSTROPHY: LAMA2

25. NEW GENES AND DISEASES

26. O.9Dominant Collagen XII-related myopathy with a distal myopathy phenotype, amenable to treatment with allele-specific knockdown

27. P.382Recessive COL12A1 loss of function EDS/myopathy overlap syndrome: confirmation and expansion of a consistently severe phenotype

30. Expanding the comprehensive national neonatal screening programme in the United Arab Emirates from 1995 to 2011

31. NEW THERAPEUTIC APPROACHES AND THEIR READOUT

32. CONGENITAL MYOPATHIES: NEMALINE AND TITINOPATHIES

33. CMT AND NEUROGENIC DISEASE

34. CONGENITAL MYOPATHIES: GENERAL AND RYR1

35. NEXT GENERATION SEQUENCING AND EXPERIMENTAL MYOLOGY

36. CONGENITAL MUSCULAR DYSTROPHIES

42. MSTO1 mutations cause mtDNA depletion, manifesting as muscular dystrophy with cerebellar involvement.

45. Intrathecal Gene Therapy for Giant Axonal Neuropathy.

46. Prevalence of iodine deficiency disorders in the United Arab Emirates measured by raised TSH levels

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