We suggest treating the Hutchinson-Gilford Progeria Syndrome by two molecules (zoledronic acid and pravastatin).The therapeutic approach which we propose has for objectives to reduce, to prevent or to delay the gravest infringements of the disease, to prolong the life of the children, and in a more general way, aim at improving their living conditions.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
15
Pravastatin : 10 mg daily Zoledronic acid : slow (30 mn) intravenous injections, diluted into 50 ml of saline solution following this schedule : * injection 1, S1: 0.0125 mg/kg of zoledronic acid * injection 2, S6: 0.025 mg/kg of zoledronic acid * injection 3, S12 and following, trimestrial basis, 0.05 mg/kg of zoledronic acid
Laboratoire de Génétique Moléculaire - Hopital de la Timone
Marseille, France
To evaluate the tolerance and efficacy of pravastatin and zoledronic acid in combination on the patient's weight, height and bone metabolism in Progeria treatment
Time frame: 48 months
To evaluate the tolerance and efficacy of the treatment on other clinical and biological symptoms
Time frame: 48 months
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