A randomized controlled study of modified cobratoxin in adrenomyeloneuropathy.
Adrenomyeloneuropathy is a peroxisomal disorder that causes demyelination, with no proven therapy. Oral modified cobratoxin was assessed in a double-blind, randomized, crossover study of eight patients. Treatment was well tolerated. There were no significant improvements with therapy. The authors do not confirm previous anecdotal reports of dramatic improvement with modified cobratoxin.
Mundy
H R
HR
Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, Queen Square, London, UK. helen.mundy@uclh.org
Jones
S J
SJ
Hobart
J C
JC
Hanna
M G
MG
Lee
P J
PJ
D000326:Adrenoleukodystrophy / Q000188:drug therapy*; D000328:Adult; D000368:Aged; D003039:Cobra Neurotoxin Proteins / Q000627:therapeutic use*; D004311:Double-Blind Method; D005071:Evoked Potentials; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D012720:Severity of Illness Index; D017211:Treatment Failure
D003039:Cobra Neurotoxin Proteins