Consensus on Gene Replacement Therapy for the Treatment for Spinal Muscular Atrophy

Members of the working group: Svetlana B. Artemyeva, MD, PhD, Head Neurological department Research Clinical Institute Pediatrics named after Yu.E. Veltishchev; Russian Federation; Elena D. Belousova, DSci, Professor, Department psychoneurology and epileptology pediatrics, Veltishchev, Dmitry V. Vlodavets, President Association Pediatric Neurologists in field myology NeoMyo, Children’s Neuromuscular Center at Surgery Yu. E. leading researcher, epileptology, Associate Professor neurology, neurosurgery medical genetics L.O. Badalyan, Faculty National Medical University N.I. Pirogov, Sergey Voronin, chief physician Genetic N.P. Bochkov, сhief freelance specialist Ministry Health Russia Far Eastern Federal District, Andrey S. Glotov, DSci., genomic medicine V.S. Baranov Obstetrics, Gynecology Reproductology D.O. Ott, Valentina I. Guzeva, Prof., children’s Neurology, Honored Scientist Federation, genetics, St. Petersburg State University, member Presidium Society Neurologists, Altynshash K. Dzhaksybaeva, Astana pediatric neurologist Republic Kazakhstan, Kazakhstan; Irina V, Zhevneronok, neurology Belarusian Academy Postgraduate Education, Belarus on hereditary neuromuscular diseases children; Republican Scientific Practical “Mother Child”, Belarus; Viktoria Kakaulina, neurologist, for Orphan Other Rare Diseases, Morozov Municipal Hospital, Lyudmila M. Kuzenkova, Child Psychoneurology, Psychoneurology Psychosomatic Pathology Health; prof. pediatrics rheumatology Sechenov First Moscow Mikhailova, Pediatrics, general Medicine Biology, Hospital Pirogov Kristina Nevmerzhitskaya, Assistant nervous diseases, Ural Sverdlovsk Region Tatyana Pervunina, Director Perinatology V.A. Almazov, Sofia G. Popovich, junior psychosomatic pathology, Health, Sosnina, Consultative Diagnostic Children, Committee Petersburg, Eugeniya Uvakina, Shchugareva, neuropathology neurosurgery, North-Western University. I.I. Mechnikov, head City Multidisciplinary Specialized High Technologies, Federation