Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

The Lancet

Volumn 388, Issue 10063, 2016, Pages 3017-3026

  Finkel, Richard S ^a   Chiriboga, Claudia A ^b   Vajsar, Jiri ^c   Day, John W ^d   Montes, Jacqueline ^b   De Vivo, Darryl C ^b   Yamashita, Mason ^e   Rigo, Frank ^e   Hung, Gene ^e   Schneider, Eugene ^e   Norris, Daniel A ^e   Xia, Shuting ^e   Bennett, C Frank ^e   Bishop, Kathie M ^e  

^a NEMOURS CHILDREN S HOSPITAL   (United States)

^b NewYork Presbyterian Hospital   (United States)

^c HOSPITAL FOR SICK CHILDREN   (Canada)

^d STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

^e ISIS PHARMACEUTICALS   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

NUSINERSEN; SURVIVAL MOTOR NEURON PROTEIN; SURVIVAL MOTOR NEURON PROTEIN 1; MESSENGER RNA; OLIGONUCLEOTIDE;

ARTICLE; ATELECTASIS; AUTOPSY; BODILY SECRETIONS; BRAIN REGION; BRAIN STEM; CLINICAL ARTICLE; CONSTIPATION; CONTROLLED STUDY; COUGHING; DIARRHEA; DRUG BLOOD LEVEL; DRUG CLEARANCE; DRUG DISTRIBUTION; DRUG DOSE INCREASE; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; DRUG UPTAKE; FEMALE; FEVER; GASTROESOPHAGEAL REFLUX; GENE DELETION; HOMOZYGOSITY; HUMAN; HUMAN CELL; HUMAN TISSUE; INFANT; INFANTILE ONSET SPINAL MUSCULAR ATROPHY; JOINT CONTRACTURE; LOADING DRUG DOSE; MALE; MOTONEURON; MOTOR PERFORMANCE; MUSCLE ACTION POTENTIAL; NERVE CELL; NEWBORN; NOSE OBSTRUCTION; OTITIS MEDIA; PERONEUS NERVE; PHASE 2 CLINICAL TRIAL; PNEUMONIA; PRIORITY JOURNAL; RASH; RESPIRATORY DISTRESS; RESPIRATORY FAILURE; RESPIRATORY TRACT INFECTION; RHINOPHARYNGITIS; RHINOVIRUS INFECTION; SCOLIOSIS; SIDE EFFECT; SPINAL CORD; SPINAL MUSCULAR ATROPHY; ULNAR NERVE; VOMITING; CLINICAL TRIAL; GENETICS; INTRASPINAL DRUG ADMINISTRATION; MUTATION; PATIENT SAFETY; SPINAL MUSCULAR ATROPHIES OF CHILDHOOD;

FEMALE; HUMANS; INJECTIONS, SPINAL; MALE; MUTATION; OLIGONUCLEOTIDES; PATIENT SAFETY; RNA, MESSENGER; SPINAL MUSCULAR ATROPHIES OF CHILDHOOD;

EID: 85006321439     PISSN: 01406736     EISSN: 1474547X     Source Type: Journal    
DOI: 10.1016/S0140-6736(16)31408-8     Document Type: Article

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