https://www.gosh.nhs.uk/our-research/our-research-infrastructure/nihr-great-ormond-street-hospital-brc/brc-news/?page=59

Biomedical Research Centre news

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

18 Dec 2015, 1:26 p.m.

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

Less intensive chemotherapy avoids irreversible side effects in some childhood cancers

18 Dec 2015, 1:24 p.m.

Children with a kidney cancer known as Wilms’ tumour, who are at low risk of relapsing, can have their chemotherapy reduced. This finding comes from a European-wide trial that studied the drug doxorubicin.

High throughput screening identifies the genetic cause underlying a rare disease in a large nationwide cohort

18 Dec 2015, 1:20 p.m.

A collaborative study between ICH and the Turkish Pediatric Endocrinology Society has used high throughput DNA analysis to identify the genetic cause of primary adrenal insufficiency in more than 80% of affected children.

A new, genetic diagnostic service for ciliopathy disorders has been launched

18 Dec 2015, 1:18 p.m.

In a collaboration between Great Ormond Street Hospital (GOSH) North-East Thames Regional Genetics and the UCL Institute of Child Health, a new genetic diagnostic service for ciliopathy disorders has been launched for service delivery to the NHS.

Funding to develop immunotherapy for childhood sarcomas

18 Dec 2015, 1:01 p.m.

Biomedical Research Centre (BRC) supported Professor John Anderson has been awarded funding from Children with Cancer UK to investigate new immunotherapy approaches for childhood sarcomas.