Study |
Intervention |
Patients |
Major adverse events |
Minor adverse events |
Bradley 1988 |
Purified bovine brain gangliosides (Cronassial), five months 40 mg Cronassial versus five months 100mg Cronassial versus placebo |
30 CMT (clinical) |
Allergic rash seen with 100mg in 2 patients, pain at injection site in one patient |
16.9% increased pain with medication versus 7.4% in the placebo group, 2.7% redness/itching versus 0.9% in placebo group, 6.8% skin rash versus 2.7% in the placebo group, tinnitus at the dose of 100mg in 2.7% versus 0.3% in the placebo group. |
Smith 2006, Chetlin 2004 |
Creatine monohydrate 5g/d combined with exercise |
39 CMT patients |
None reported. |
Mild gastrointestinal upset, leg cramps, fatigue, delayed‐onset soreness. |
Sahenk 2005 |
Neurotrophin‐3 starting with 50 µg/kg increasing up to 150 µg/kg |
4 CMT1A (genetically proven) |
None reported. |
Irritation at injection site, mild diarrhoea. |
Prensky 1984 |
Methylprednisolone 40 to 60 mg/m2 |
14 CMT clinical |
Weight gain of 15 to 18 pounds in 3 patients, proximal myopathy and psychiatric illness in 1 patient (drop out), compression fracture of dorsal vertebrae T‐12 due to significant worsening of osteoporosis in 1 patient, severe leg cramps in 1 patient, pseudotumour when steroid was stopped in 1 patient, depression in 1 patient, hypertrophic nerve root in one patient. |
None reported. |
Russell 1995 |
3,4‐diaminopyridine (3,4‐DAP) 4x5mg/day up to 4x20mg/day |
27 CMT patients (clinical) |
None reported. |
71% facial pain and predominantly perioral parasthesia with a maximum duration of 90 minutes, 29% muscle fatigue, 18% nauseas and abdominal discomfort, 15% insomnia, 12% transient urinary frequency, 6% increased tearing or nasal dicharge, 6% facial flushing, 6% transient hand tremor or muscle twitching, 6% headache, 6% transient parageusia. |
Dyck 1982a, Dyck 1982b |
Prednisone |
Single cases |
None reported. |
None reported. |
Folkers 1985, Folkers 1995 |
Q10 |
Single cases |
None reported. |
None reported. |
Lisett 1998 |
Growth hormone replacement |
Single case |
None reported. |
None reported. |
Jaradeh 1992 |
Levodopa |
6 unspecified CMT cases. |
None reported. |
None reported. |
Williams 1986 |
Vitamin E combined with Cis‐linolein, gamma‐linoleic, oleic acid |
20 CMT patients (clinical). |
None reported. |
None reported. |