US FDA approves world's most expensive drug Zolgensma one-time treatment for SMA

Swiss drugmaker Novartis has received US approval for its spinal muscular atrophy gene therapy Zolgensma® (onasemnogene abeparvovec-xioiT) for the treatment of pediatric patients less than 2years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene). The one time treatment drug is priced at a record $2.125m. Novartis executives have defended the price, saying a one-time treatment is more valuable than expensive long-term treatments that cost several hundred thousand dollars a year. Zolgensma® is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to...
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Global Burden of Disease Fellowship Programme

Applications are invited from interested Project Supervisors for Seed Grant Funding under the Global Burden of Disease ( GBOD ) Research Trainee Fellowship program a new initiative of the International Child Neurology Association (ICNA). The Award The award consists of $ 2,000 for room and board for ~ 2-3 months plus up to $ 2,000 for roundtrip airfare. Timeline The deadline date for receipt of proposals on open competition from project supervisors is August 1. The Scoring and Selection of Grants fulfilling Criteria by Research Task Force Committee (10 point scoring system) will be completed by September 1 and results announced on ICNApedia. The deadline for...
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Herman Doose [1927-2018]

Professor Hermann Doose, whose name is entwined with Myoclonic Astatic Epilepsy otherwise known as “Doose Syndrome” passed away on April 23rd, 2018 following a brief illness. He was 90yrs old. Professor Doose was a founding member of the neuropediatric society in Germany.Hermann Doose was born on September 1927 in Lübeck, North Germany. His father was a surgeon and mother a gynaecologist. At the age of 16 he was recruited to the army during the last year of the war. A subsequent injury and admission to hospital resulted in him being the lone survivor of the group.  Doose studied medicine at the...
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Did Leonardo da Vinci have ADHD?

Leonardo da Vinci produced some of the world’s most iconic art, but historical accounts of his work practices and behaviour show that he struggled to complete projects. Drawing on these accounts, Professor Catani lays out the evidence supporting his hypothesis that, as well as explaining his chronic procrastination, ADHD could have been a factor in Leonardo’s extraordinary creativity and achievements across the arts and sciences. Professor Catani, from the Institute of Psychiatry, Psychology & Neuroscience at King’s, says: ‘While impossible to make a post-mortem diagnosis for someone who lived 500 years ago, I am confident that ADHD is the most convincing...
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