Adult Medical Issues in Cystinosis--William Gahl, M.D.

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- Approved August 15, 1994- Approved for pre-transplantpatients only- Cost remains reasonable: ~$3000-$5000/year in U.S.
Pre-transplant, oral cysteaminetherapy:- Preserves renal function- Allows for a normal growth rate- Preserves thyroid function- Depletes muscle & liver of cystine
Page 1 and 2: Section on Human Biochemical Geneti
Page 3 and 4: CYSTINOSIS NATURAL HISTORYAgeBirthI
Page 5 and 6: CYSTINOSIS NATURAL HISTORYPost-Tran
Page 7 and 8: 22-year old withcystinosis,myopathy
Page 9 and 10: Cystine crystalsin muscleCystine cr
Page 11 and 12: Swallowing DifficultyPooling invall
Page 13 and 14: Coronary Artery CalcificationCorona
Page 15 and 16: Cerebral atrophyin a 24-year oldman
Page 17 and 18: Gastric varixDuodenal varix
Page 19 and 20: 100 Nephropathic CystinosisAdults (
Page 21 and 22: Adult Cystinosis Patients(NIH, 1986
Page 25 and 26: NIH Intent-to-treat Analysis forOra
Page 29 and 30: 4Muscle Cystine321No Cysteamine010
Page 31: CYSTEAMINE EYEDROPSUntreated Treate
Page 35 and 36: Post-Transplant CystinosisComplicat
Page 37 and 38: 101 Cystinosis Patients4.54Swallowi
Page 39 and 40: 60%50%n=1040%n=3830%% OF PATIENTS W
Page 41 and 42: 100%90%n=28n=10% OF PATIENTS WITH P
Page 43 and 44: 100 Adult Cystinosis PatientsCystea
Page 45 and 46: 100 Adult Cystinosis PatientsCholes
Page 47 and 48: Effect of Oral Cysteamine onthe Lat
Page 52 and 53: Nephrogenic FibrosingDermopathy
Page 54 and 55: NFD: More than skin deep• Patholo
Page 56 and 57: June 2006 (25 cases); updated Decem
Page 58: Risk Mitigation Strategy• Elimina
Page 61 and 62: Transmission EMof conjunctival cell
Page 63 and 64: N=89 N=32 N=24 N=9100%90%80%70%60%5

Adult Medical Issues in Cystinosis--William Gahl, M.D.

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