871 results on '"Friedreich ataxia"'
Search Results
2. Friedreich Ataxia
3. Friedreich Ataxia Caregiver-Reported Health Index
4. An RNA-seq study in Friedreich ataxia patients identified hsa-miR-148a-3p as a putative prognostic biomarker of the disease
5. Deciphering the ferroptosis pathways in dorsal root ganglia of Friedreich ataxia models. The role of LKB1/AMPK, KEAP1, and GSK3beta in the impairment of the NRF2 response
6. Characterization of clinical serum cardiac biomarker levels in individuals with Friedreich ataxia
7. Pharmacotherapeutic strategies for Friedreich Ataxia: a review of the available data
8. The MOXIe Trial of Omaveloxolone in Friedreich Ataxia: Exploring the Transient Nature of Treatment-emergent Adverse Events (P7-3.016)
9. Approval of omaveloxolone for Friedreich ataxia
10. Leber Hereditary Optic Neuropathy in 2 Sisters With Friedreich Ataxia
11. Accelerometer-based measures in Friedreich ataxia: a longitudinal study on real-life activity
12. Characterization of Cardiac-Onset Initial Presentation in Friedreich Ataxia
13. A Pilot Phase 2 Randomized Trial to Evaluate the Safety and Potential Efficacy of Etravirine in Friedreich Ataxia Patients
14. Content validation of the modified Friedreich Ataxia Rating Scale-Activities of Daily Living (mFARS-ADL) for use with Gaucher disease type 3 patients: A qualitative study
15. Friedreich Ataxia
16. Omaveloxolone for the treatment of Friedreich ataxia: clinical trial results and practical considerations
17. Correction: Human frataxin, the Friedreich ataxia deficient protein, interacts with mitochondrial respiratory chain
18. Tissue Iron in Friedreich Ataxia
19. Omaveloxolone: a groundbreaking milestone as the first FDA-approved drug for Friedreich ataxia
20. Mitochondrial impairment, decreased sirtuin activity and protein acetylation in dorsal root ganglia in Friedreich Ataxia models
21. Human frataxin, the Friedreich ataxia deficient protein, interacts with mitochondrial respiratory chain
22. PCR139 'We Have Conversations That Other Couples Don′t Have to Have:' A Qualitative Study of the Impact of Caregiving in Friedreich Ataxia (FA)
23. CO142 A Retrospective Study Characterizing Age at Key Clinical Disease Indicators Among Patients With Friedreich Ataxia Using Health Administrative Claims Data in the United States
24. Developing an optimal mouse model for Friedreich ataxia
25. Clinical stage and plasma neurofilament concentration in adults with Friedreich ataxia
26. PCR84 The Daily Life and Psychosocial Impacts of Friedreich Ataxia: A Qualitative Study of Patient Lived Experiences
27. Friedreich ataxia
28. CO105 A Retrospective Study Characterizing Age at Loss of Ambulation Among Patients With Friedreich Ataxia Using Health Administrative Claims Data in the United States
29. Effectiveness of rehabilitation intervention in persons with Friedreich ataxia
30. Calcitriol treatment is safe and increases frataxin levels in Friedreich Ataxia patients.
31. Correction to Safety and Efficacy of Omaveloxolone in Friedreich Ataxia (MOXIe Study)
32. Proprioceptive and tactile processing in individuals with Friedreich ataxia: an fMRI study
33. Patient-derived iPSC models of Friedreich ataxia: a new frontier for understanding disease mechanisms and therapeutic application
34. Propensity matched comparison of omaveloxolone treatment to Friedreich ataxia natural history data
35. Rationale and protocol of a double-blind, randomized, placebo-controlled trial to test the efficacy, safety, and tolerability of dimethyl fumarate in Friedreich Ataxia (DMF-FA-201)
36. TEMPORARY REMOVAL: Omaveloxolone approved for patients aged 16 years and older with Friedreich ataxia (FRDA): A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG)
37. Finding an Appropriate Mouse Model to Study the Impact of a Treatment for Friedreich Ataxia on the Behavioral Phenotype
38. A Spotlight on Friedreich Ataxia: Optimising the Patient Journey from Diagnosis to Disease Management
39. Patient-reported, health economic and psychosocial outcomes in patients with Friedreich ataxia (PROFA): protocol of an observational study using momentary data assessments via mobile health app
40. Friedreich ataxia
41. Mitochondrial Dysfunction in Friedreich Ataxia
42. A Bayesian Network Approach for Friedreich Ataxia Severity Classification using Probability Modelling
43. A Novel Feature from Instrumented Utensils for Clinical Assessment of Friedreich Ataxia
44. Patient-specific iPSCs Reveal Vascular Dysfunction in Friedreich Ataxia
45. Neuropsychiatric symptoms in spinocerebellar ataxias and Friedreich ataxia
46. Retinal hypoplasia and degeneration result in vision loss in Friedreich ataxia
47. N°234 – Multimodal evoked fields in Friedreich ataxia
48. A Milestone in the Treatment of Ataxias: Approval of Omaveloxolone for Friedreich Ataxia
49. Diabetic ketoacidosis as first presentation of diabetes mellitus in a subject with Friedreich' ataxia. A case report and review of literature
50. Short-read genome sequencing allows ‘en route’ diagnosis of patients with atypical Friedreich ataxia
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