Doctors monitor Real-World impact of SMA drug in adults
NCT ID NCT04159987
Summary
This study is observing how the approved drug Spinraza (nusinersen) affects adults living with SMA type II, a genetic muscle-weakening condition. Researchers are closely tracking 20 wheelchair-using adults over more than two years to see how their motor skills, strength, breathing, and quality of life change while on the treatment. The goal is to gather detailed, real-world data on the drug's long-term effects in this specific adult population.
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Contacts and locations
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Locations
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APHP
Paris, Île-de-France Region, 94000, France
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CHRU de Lille
Lille, Hauts-de-France, 59000, France
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CHU de Lyon
Lyon, Auvergne-Rhône-Alpes, 69677, France
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CHU de Montpellier
Montpellier, Occitanie, 34090, France
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CHU de Nice
Nice, Provence-Alpes-Côte d'Azur Region, 06000, France
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CHU de Toulouse
Toulouse, Occitanie, 31059, France
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Hopital de la Timone - APHM
Marseille, Provence-Alpes-Côte d'Azur Region, 13005, France
Conditions
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