Showing posts with label drug_therapy. Show all posts
Showing posts with label drug_therapy. Show all posts
Thursday, 5 April 2018
Prescribing antipsychotic medication for children with autism
A new study has suggested that children with intellectual difficulty or
autism are more likely to be given antipsychotic medication from a
younger age than those without intellectual disability and have higher
rates of hospitalisation for depression and for injury and also are at
risk of other medical side effects. Click here to read further.
Tuesday, 13 February 2018
Drug company says it will negotiate terms to allow children to access new treatment for rare genetic disorder
Drug company says it will negotiate terms to allow children to access new treatment for rare genetic disorder.
This preliminary decision, which is now the subject of public consultation until 5 March, comes as the company confirmed it is to negotiate with NHS England terms that could allow the drug to be made available while uncertainties in the evidence of its long term benefits are addressed.
Children with CLN2, which is also known as Batten Disease, live to between 8 years and early adolescence; the average life expectancy is 10 years. It is estimated that in the UK there are around 30 to 50 children living with the condition.
CLN2 is a progressive condition caused by the deficiency of the enzyme tripeptidyl peptidase 1. This results in the abnormal storage of proteins and lipids in neurons and other cells, preventing them from functioning normally.
Symptoms in children with CLN2 begin from around the age of 2 and can then progress rapidly with the onset of seizures, decline in speech, loss of mobility, involuntary muscle spasms, progressive dementia and visual impairment leading to blindness.
There is currently no cure or life-extending treatments for CLN2 and clinical management is limited to symptom relief and supportive and palliative care.
Costing over £500,000 for each year’s treatment, cerliponase alfa is an enzyme replacement therapy administered directly into the brain via a surgically implanted permanent access device.
See the full details from NICE at https://www.nice.org.uk/news/article/drug-company-says-it-will-negotiate-terms-to-allow-children-to-access-new-treatment-for-rare-genetic-disorder
This preliminary decision, which is now the subject of public consultation until 5 March, comes as the company confirmed it is to negotiate with NHS England terms that could allow the drug to be made available while uncertainties in the evidence of its long term benefits are addressed.
Children with CLN2, which is also known as Batten Disease, live to between 8 years and early adolescence; the average life expectancy is 10 years. It is estimated that in the UK there are around 30 to 50 children living with the condition.
CLN2 is a progressive condition caused by the deficiency of the enzyme tripeptidyl peptidase 1. This results in the abnormal storage of proteins and lipids in neurons and other cells, preventing them from functioning normally.
Symptoms in children with CLN2 begin from around the age of 2 and can then progress rapidly with the onset of seizures, decline in speech, loss of mobility, involuntary muscle spasms, progressive dementia and visual impairment leading to blindness.
There is currently no cure or life-extending treatments for CLN2 and clinical management is limited to symptom relief and supportive and palliative care.
Costing over £500,000 for each year’s treatment, cerliponase alfa is an enzyme replacement therapy administered directly into the brain via a surgically implanted permanent access device.
See the full details from NICE at https://www.nice.org.uk/news/article/drug-company-says-it-will-negotiate-terms-to-allow-children-to-access-new-treatment-for-rare-genetic-disorder
Labels:
drug_therapy,
genetics,
paediatrics,
xMH
Monday, 5 February 2018
UHCW publication: subtle tardive dyskinesia with paliperidone palmitate
The importance of clinical observation: A case of subtle tardive dyskinesia with paliperidone palmitate.
Omer, Hisham; Thompson, Andrew D
Aust N Z J Psychiatry. 2018 Jan 1:4867417750758. doi: 10.1177/0004867417750758.[letter - no abstract available]
Omer, Hisham; Thompson, Andrew D
Aust N Z J Psychiatry. 2018 Jan 1:4867417750758. doi: 10.1177/0004867417750758.[letter - no abstract available]
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