Search Results

Treatment of the central nervous system as well as the peripheral organs proves beneficial in severe cases of Spinal Muscular Atrophy (SMA)

A pre-clinical study investigating treatment options for a severe form of Spinal Muscular Atrophy (SMA) has demonstrated that optimal treatment of a morpholino antisense oligonucleotide drug is achieved when the drug reaches the central nervous system as well as the peripheral organs.

Genetic aspects of primary immunodeficiency

This booklet has been produced jointly between PID UK, Great Ormond Street Hospital (GOSH) and the Great North Children’s Hospital. It is designed to help answer the questions that families may have about the genetic aspects of primary immunodeficiencies (PID). The information has been reviewed by the PID UK Medical Advisory Panel and Patient Representative Panel and by families affected by PID but should not replace advice from a clinical immunologist or a geneticist.

Richard Scott

Richard Scott attended medical school at Cambridge University and University College London before training in Paediatrics and subsequently Clinical Genetics in London.