bsp;
NHSC/NIHR http://www.hsc.nihr.ac.uk/topics/ataluren-for-nonsense-mutation-dystrophinopathy/
References
Available only to registered users
Category
BNF Category: Drugs used in neuromuscular disorders (10.02)
Pharmacology: Dystrophin stimulant
Epidemiology: The incidence of DMD is approximately 1 in 3,600-6,000 male births per year. In the UK, there are approximately 100 boys diagnosed with DMD each year & at any one time there are about 1,500 known to have the disease. Approximately 10-15% of patients have a nonsense mutation, which equates to between 150 and 195 pts in the UK [NHSC review].
Indication: Duchenne muscular dystrophy
Method(s) of Administration
Oral
Company Information
Name: PTC Therapeutics
US Name: PTC Therapeutics
Further Information
Anticipated commissioning route (England) NHSE
High cost drug list? Yes
Tariff Not routinely commissioned by NHSE - IFR approval [28]
In NICE timetable: Yes
When: Feb / 2016
Note: www.nice.org.uk/guidance/indevelopment?type=hst
Implications Available only to registered users
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