New SMA drug shows promise in major safety trial
NCT ID NCT03032172
Summary
This study tested the safety and how the body processes the drug risdiplam in 174 adults and children with spinal muscular atrophy (SMA), a genetic muscle-wasting disease. All participants had previously tried other SMA treatments. The main goal was to see if risdiplam was safe and well-tolerated, while also checking if it increased levels of a crucial protein missing in SMA patients.
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Contacts and locations
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Locations
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Birmingham Heartlands Hospital
Birmingham, B9 5SS, United Kingdom
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Boston Childrens Hospital
Boston, Massachusetts, 02115, United States
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CHRU de Montpellier, Hopital Gui de Chauliac
Montpellier, 34295, France
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Columbia University Medical Center
New York, New York, 10032, United States
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Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Lombardy, 20122, Italy
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Hopital Femme Mere Enfant
Bron, 69677, France
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Hopital Roger Salengro
Lille, 59037, France
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Hopital des Enfants
Toulouse, 31059, France
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Hôpital Necker-Enfants Malades
Paris, 75015, France
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IRCCS Istituto Giannina Gaslini
Genoa, Liguria, 16147, Italy
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IRCCS Ospedale Pediatrico Bambino Gesù
Rome, Lazio, 00165, Italy
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Klinika Neurologii I Wydzialu Lekarskiego WUM w Warszawie
Warsaw, 02-097, Poland
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Nemours Children's Hospital
Orlando, Florida, 32827, United States
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Policlinico Agostino Gemelli
Rome, Lazio, 00168, Italy
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Stanford University Medical Center
Palo Alto, California, 94304, United States
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The Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, NE1 4LP, United Kingdom
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UCL Institute of Child Health & Great Ormond Street Hospital for Children
London, WC1N 1EH, United Kingdom
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UMC Utrecht
Utrecht, 3584 CX, Netherlands
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UOSD Malattie Neurodegenerative
Messina, Sicily, 98125, Italy
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UZ Gent
Ghent, 9000, Belgium
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UZ Leuven Gasthuisberg
Leuven, 3000, Belgium
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Universitäts-Kinderspital (UKBB) Neuropädiatrie
Basel, 4005, Switzerland
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Universitätsklinikum Essen
Essen, 45147, Germany
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Universitätsklinikum Freiburg
Freiburg im Breisgau, 79106, Germany
Conditions
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