Emerging therapies and challenges in spinal muscular atrophy

Annals of Neurology

Volumn 81, Issue 3, 2017, Pages 355-368

  Farrar, Michelle A ^a   Park, Susanna B ^b   Vucic, Steve ^c   Carey, Kate A ^a   Turner, Bradley J ^d   Gillingwater, Thomas H ^e   Swoboda, Kathryn J ^f   Kiernan, Matthew C ^b  

^a UNIVERSITY OF NEW SOUTH WALES   (Australia)

^b UNIVERSITY OF SYDNEY   (Australia)

^c UNIVERSITY OF SYDNEY   (Australia)

^d UNIVERSITY OF MELBOURNE   (Australia)

^e UNIVERSITY OF EDINBURGH   (United Kingdom)

^f MASSACHUSETTS GENERAL HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

4 (1 AMINOETHYL) N (4 PYRIDYL)CYCLOHEXANECARBOXAMIDE; 4 PHENYLBUTYRIC ACID; ADENO ASSOCIATED VIRUS VECTOR; ANTISENSE OLIGONUCLEOTIDE; AVXS 101; BAY 559837; BETA CATENIN; BRANAPLAM; CK 2127107; FAMPRIDINE; FASUDIL; FLAVONOID; GENE THERAPY AGENT; GROWTH HORMONE; HYDROXYUREA; LEVACECARNINE; NEUROPROTECTIVE AGENT; NUSINERSEN; OLESOXIME; PYRIDOSTIGMINE; QUERCETIN; RG 7916; RILUZOLE; STL 182; SURVIVAL MOTOR NEURON PROTEIN 2; UNCLASSIFIED DRUG; VALPROIC ACID; VIRUS VECTOR;

CLINICAL TRIAL (TOPIC); DISEASE CLASSIFICATION; GASTROINTESTINAL DISEASE; GENE REPLACEMENT THERAPY; GENE TRANSFER; HUMAN; KINESIOTHERAPY; MENTAL DISEASE; MOTOR PERFORMANCE; MUSCULOSKELETAL DISEASE; NEUROPROTECTION; NEWBORN SCREENING; NONHUMAN; NUTRITIONAL DISORDER; NUTRITIONAL SUPPORT; ONSET AGE; PATHOGENESIS; PATIENT CARE; PATIENT MONITORING; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; PROGNOSIS; REHABILITATION CARE; RESPIRATORY TRACT DISEASE; REVIEW; SPINAL MUSCULAR ATROPHY; THERAPEUTIC INDEX; ANIMAL; GENETICS; MUSCULAR ATROPHY, SPINAL;

ANIMALS; HUMANS; MUSCULAR ATROPHY, SPINAL;

EID: 85013421972     PISSN: 03645134     EISSN: 15318249     Source Type: Journal    
DOI: 10.1002/ana.24864     Document Type: Review

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