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New 36-Month Apitegromab Extension Data Reinforce Long-Term Substantial and Sustained Improvement of Motor Function in Phase 2 TOPAZ Trial Patients with Nonambulatory Spinal Muscular Atrophy

New 36-Month Apitegromab Extension Data Reinforce Long-Term Substantial and Sustained Improvement of Motor Function in Phase 2 TOPAZ Trial Patients with Nonambulatory Spinal Muscular Atrophy
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Four-year follow up data for Roche's Evrysdi show continued increase in number of children with a severe form of spinal muscular atrophy (SMA) able to sit, stand and walk

Data from ongoing FIREFISH study confirm long-term efficacy and safety profile of Evrysdi in children with Type 1 SMANinety-one percent of children were alive at month 48More than 95% maintained the. ....

United Kingdom , United States , City Of , Kirti Pandey , Loren Kalm , Levi Garraway , Birgit Masjost , Nathalie Altermatt , Bruno Eschli , Gerard Tobin , Sabine Borngr , Giovanni Baranello , British Pharmacological Society , Roche Group , Toddler Development Third Edition , Drug Administration , Research Clinical Care Meeting , Head Of Global Product Development , Society For Medicines Research , Office Of Orphan Products Development , European Medicines Agency , Roche Group Media Relations , Institute Of Child Health Hospital , Gross Motor Scale , Bayley Scales , Hammersmith Infant Neurological Examination ,

F. Hoffmann-La Roche Ltd: Four-year follow up data for Roche's Evrysdi show continued increase in number of children with a severe form of spinal muscular atrophy (SMA) able to sit, stand and walk

F. Hoffmann-La Roche Ltd: Four-year follow up data for Roche's Evrysdi show continued increase in number of children with a severe form of spinal muscular atrophy (SMA) able to sit, stand and walk
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United States , United Kingdom , City Of , Sabine Borngr , Bruno Eschli , Gerard Tobin , Nathalie Altermatt , Kirti Pandey , Giovanni Baranello , Birgit Masjost , Levi Garraway , Loren Kalm , Research Clinical Care Meeting , Roche Group , Head Of Global Product Development , Office Of Orphan Products Development , Roche Group Media Relations , British Pharmacological Society , Toddler Development Third Edition , Society For Medicines Research , Drug Administration , European Medicines Agency , Institute Of Child Health Hospital , Gross Motor Scale , Bayley Scales , Hammersmith Infant Neurological Examination ,

Biogen Inc.: New Data at Cure SMA Highlight Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs After Gene Therapy

Biogen Inc.: New Data at Cure SMA Highlight Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs After Gene Therapy
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New Data at Cure SMA Highlight Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs After Gene Therapy

New Data at Cure SMA Highlight Potential Benefit of SPINRAZA® (nusinersen) in Infants and Toddlers with Unmet Clinical Needs After Gene Therapy
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United Kingdom , Maha Radhakrishnan , Chuck Triano , Jack Cox , Ionis Pharmaceuticals Inc , Biogen Inc , Research Clinical Care Meeting , Exchange Commission , Drug Administration , Clinical Care Meeting , Chief Medical Officer , Clinical Outcomes , Crystal Proud , Pediatric Neurologist , Analysis Evaluating Real World Impact , Novel Device Aimed , Enhancing Treatment Experience , Alcyone Therapeutics , Investigational Device Exemption , Ionis Pharmaceuticals , Important Safety , Private Securities Litigation Reform Act ,