Shire Enhances its Orphan Drug Pipeline With the Acquisition of a New Clinical Candidate for Metachromatic Leukodystrophy

BASINGSTOKE, England and CAMBRIDGE, Massachusetts, April 24 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announces the acquisition of arylsulfatase -A (ASA) an Enzyme Replacement Therapy (ERT) in Phase 1-2 clinical trials for Me...

AIMSPRO is Awarded TGA Orphan Status for the Treatment of Amyotrophic Lateral Sclerosis

...mon form of Motor Neuron Disease (MND), also known as Lou Gehrig's disease. AIMSPRO has already achieved TGA Orphan Status for the treatment of Krabbe leukodystrophy (Krabbe's disease). Daval is now evaluating an international, multi-centre, placebo controlled, Randomised Clinical Trial to determine whether later s...

Leukodystrophy in Medical Definition

Pathology

... GM1 gangliosidoses - Mucolipidosis type IV - Gaucher's disease - Niemann-Pick disease - Farber disease - Fabry's disease - Metachromatic leukodystrophy - Krabbe disease Neuronal ceroid lipofuscinosis ( Batten disease ) - Cerebrotendineous xanthomatosis - Cholesteryl ester storage disease ( W...

Leukodystrophy in Medical Dictionary

Leukodystrophy

Leukodystrophy refers to a group of disorders characterized by progressive degeneration of the white matter of the brain. The leukodystrophies are caused by imperfect growth or development of the myelin sheath, the fatty covering that acts as an insulator around nerve... leukodystrophy ( ...

Krabbe disease

...ine resources about Krabbe disease . United leukodystrophy Foundation: The leukodystrophies are genetically d...ty of Maryland Medical Center. globoid cell leukodystrophy ( ) n. An inherited metabolic encephalopathy of i... Krabbe disease , also called globoid cell leukodystrophy (GCL), is a rare, ... Type 1, or infantile Krabb...

Canavan

...isease, one of the most common cerebral degenerative diseases of ... Well, hello there! Welcome to Trudi Canavan's Website Index. ... United leukodystrophy Foundation: The leukodystrophies are genetically determined progressive disorders that affect the brain, spinal cord and peripheral nerves. ... ...

Adrenoleukodystrophy

...rview, X-linked adrenoleukodystrophy describes several ... Adrenoleukodystrophy is passed down from parents to their children as an ... United leukodystrophy Foundation: The leukodystrophies are genetically determined progressive disorders that affect the brain, spinal cord and peripheral nerves. ... ...

Leukodystrophy in Biological Technology

Shire's New Product Portfolio Delivers Strong Quarterly Performance

...f Shire's US development plan and to strengthen the chances of regulatory and commercial success in the US. METAZYM (HGT-1111) - Metachromatic leukodystrophy ("MLD") - Shire has an ongoing enzyme replacement therapy program for the treatment of MLD, which is a lysosomal storage disorder that results ...

Shire Delivers Strong Quarter: Driven by $243m of New Product Sales (+164% YOY Growth). Revenue Guidance Upgrade.

...dies. It is anticipated that this development program will support global filings in the second half of 2009. METAZYM (HGT-1111) - Metachromatic leukodystrophy ("MLD") - METAZYM is being investigated for the treatment of MLD and has completed a Phase 1b clinical trial in twelve patients in Europe and an ...

Following a Good Start to the Year Shire Reaffirms Full Year 2008 Financial Guidance

...global rights to the clinical candidate arylsulfatase-A ("ASA"), currently known as METAZYM(TM), being investigated for the treatment of Metachromatic leukodystrophy ("MLD"). MLD is caused by a deficiency in the enzyme ASA which causes an excess concentration of sufatide in cells and an ensuing breakdown of myelin....