Data from EMA (European Medicines Agency) - Curated by EPG Health - Last updated 12 January 2018
Indication(s)
Learning Zones
An epgonline.org Learning Zone (LZ) is an area of the site dedicated to providing detailed self-directed medical education about a disease, condition or procedure.
Migraine Knowledge Centre
The Migraine Knowledge Centre features latest research on the prevalence and impact of migraine, the proposed neurological basis of the condition (and how this is being translated into new and exciting drug therapies), as well as current patient care strategies collated from headache organisations worldwide.
Acute and Advanced Heart Failure
What are the most effective treatments for acute heart failure? Can you define advanced heart failure? Discover here...
+ 3 more
Fabry Disease
Explore the pathophysiology and treatment options for Fabry disease, a deficiency of the lysosomal enzyme alpha-galactosidase A
Hyperammonaemia
Hyperammonaemia can result in serious neurological damage or death. Could you recognise the signs and symptoms?
+ 2 more
Cushing's Syndrome
Cushing’s syndrome shares symptoms such as hypertension, glucose intolerance and obesity with other common conditions – how can you confidently diagnose this rare disorder?
+ 2 more
Cystic Fibrosis
View disease awareness information, treatment options and European Cystic Fibrosis Society best practice guidelines.
+ 1 more
Pain, Agitation and Delirium (PAD) Management
Discover the latest guidance on assessment and management of pain, agitation and delirium for patients requiring sedation.
+ 4 more
Related Content
A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
The Phase 1/2 study (190-201) evaluated the efficacy and safety of doses up to 300 mg/every other week (qow) BMN 190 in patients with CLN2. The dose and regimen for this study (190-202) are based on the results of the 190-201 study.
Added 9 months ago
A Safety, Tolerability, and Efficacy Study of Intracerebroventricular BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
This Phase 2 open-label, multicenter study will evaluate the safety, tolerability, and efficacy of BMN 190 intracerebroventricular (ICV) administration every other week (qow) for a period of 96 weeks, in patients with CLN2.
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A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (ENDEAR)
The primary objective of the study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally (IT) to participants with infantile-onset SMA.
Added 9 months ago
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Is amyotrophic lateral sclerosis/frontotemporal dementia an autophagy disease?
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Advances, challenges and future directions for stem cell therapy in amyotrophic lateral sclerosis.
Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative condition where loss of motor neurons within the brain and spinal cord leads to muscle atrophy, weakness, paralysis and ultimately death...
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The heat shock response in neurons and astroglia and its role in neurodegenerative diseases.
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Association Between Motor Symptoms and Brain Metabolism in Early Huntington Disease.
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Therapies targeting DNA and RNA in Huntington's disease.
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EFNS guidelines on the Clinical Management of Amyotrophic Lateral Sclerosis (MALS) – revised report of an EFNS task force
Objectives: To provide evidence-based or expert recommendations for the diagnosis and management of ALS based on a literature search and the consensus of an expert panel.
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Use of antibody testing in nervous system disorders
To evaluate service provision and quality assurance schemes for clinically useful autoantibody tests in neurology.
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EFNS guidelines on diagnosis and treatment of primary dystonias
Primary dystonias are chronic and often disabling conditions with a widespread spectrum mainly in young people.
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More information
Category | Value |
---|---|
Agency product number | EMEA/H/C/004312 |
Orphan designation | Yes |
Date First Approved | 30-05-2017 |
Type | Medicinal product subject to restricted medical prescription |
Marketing authorisation holder | Biogen Idec Ltd |