Prognostic factors and treatment-effect modifiers in spinal muscular atrophy [PDF]
Spinal muscular atrophy (SMA) is a rare, progressive neuromuscular disease characterized by loss of motor neurons and muscle atrophy. Untreated infants with Type 1 SMA do not achieve major motor milestones, and death from respiratory failure typically ...
Baranello, G+9 more
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Effective local treatment of necrotizing fasciitis using a chlorine solution obtained by electrolysis [PDF]
Introduction. Spinal muscular atrophy (SMA) and acute lymphoblastic leukemia (ALL) are rare diseases, with usual onset in childhood. To date, no cases have been reported where these conditions co-exist in one patient.
Kovačević Predrag T.
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Serum creatine kinase and creatinine in adult spinal muscular atrophy under nusinersen treatment [PDF]
Maren Freigang+20 more
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B.06 Safety and efficacy of nusinersen in infants/children with spinal muscular atrophy (SMA): part 1 of the phase 2 EMBRACE study [PDF]
PB Shieh+13 more
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Nusinersen in type 0 spinal muscular atrophy: should we treat? [PDF]
Eloisa Tiberi+9 more
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Nusinersen treatment of spinal muscular atrophy: current knowledge and existing gaps [PDF]
Teresa Gidaro, Laurent Servais
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Administration of nusinersen via paramedian approach for spinal muscular atrophy [PDF]
Hideyuki Iwayama+6 more
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Intrathecal Administration of Nusinersen Using the Ommaya Reservoir in an Adult with 5q-Related Spinal Muscular Atrophy Type 1 and Severe Spinal Deformity [PDF]
Vasileios Papaliagkas+9 more
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Spinal muscular atrophy and acute lymphoblastic leukemia - is it just a coincidence? [PDF]
Introduction. Spinal muscular atrophy (SMA) and acute lymphoblastic leukemia (ALL) are rare diseases, with usual onset in childhood. To date, no cases have been reported where these conditions co-exist in one patient.
Jovanović Kristina+4 more
doaj +1 more source
Preliminary Safety and Tolerability of a Novel Subcutaneous Intrathecal Catheter System for Repeated Outpatient Dosing of Nusinersen to Children and Adults With Spinal Muscular Atrophy [PDF]
Kevin A. Strauss+10 more
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