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| ID | Type | Description | Link |
|---|---|---|---|
| 2008-15 |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 1 | Other | Zoledronic acid, pravastatin |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Zoledronic acid, pravastatin | Drug | Pravastatin : 10 mg daily Zoledronic acid : slow (30 mn) intravenous injections, diluted into 50 ml of saline solution following this schedule :
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 | 48 months |
| Measure | Description | Time Frame |
|---|---|---|
| To evaluate the tolerance and efficacy of the treatment on other clinical and biological symptoms | 48 months |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Nicolas LEVY, MD | Assistance Publique des Hopitaux de Marseille | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Laboratoire de Génétique Moléculaire - Hopital de la Timone | Marseille | 13385 | France |
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| ID | Term |
|---|---|
| D011371 | Progeria |
| ID | Term |
|---|---|
| D000083083 | Laminopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008661 | Metabolism, Inborn Errors |
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| ID | Term |
|---|---|
| D000077211 | Zoledronic Acid |
| D017035 | Pravastatin |
| ID | Term |
|---|---|
| D004164 | Diphosphonates |
| D063065 | Organophosphonates |
| D009943 | Organophosphorus Compounds |
| D009930 | Organic Chemicals |
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|
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D007093 |
| Imidazoles |
| D001393 | Azoles |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D009281 | Naphthalenes |
| D011084 | Polycyclic Aromatic Hydrocarbons |
| D006841 | Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D011083 | Polycyclic Compounds |