Leukodystrophy

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

... Replacement Therapy (ERT) in Phase 1-2 clinical trials for Metachromatic leukodystrophy (MLD) from the Danish company Zymenex A/S (Zymenex). MLD is a serious, ... Scriver et al 1995 Notes to editors Disease Background Metachromatic leukodystrophy (MLD) is in the family of lysosomal storage diseases (LSD's). MLD is an ...

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

... 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 ...

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 ( ...

Leukodystrophy in Medical Dictionary

Leukodystrophy

... Adreno leukodystrophy Dr Trisha Macnair Adreno leukodystrophy (ALD) is a rare inherited disorder that leads ... death . Take a look at the causes. Adreno leukodystrophy describes any of several closely related ...

Cml

... - chronic lymph ocytic (CLL) Leukemia - hairy cell Leukemia - nonlymphocytic (ANLL) Leukemic reticuloendotheliosis Leukemoid reaction leukodystrophy - metachromatic Leukoplakia Lewy body dementia ... Previously approved for the treatment of patients with chronic myeloid leukemia ( ...

Metabolic Diseases

... sugar found in milk ( lactose ) can lead to accumulation of another sugar called galactitol, ... Storage or metabolic diseases Ad renal leukodystrophy (ALD) Gaucher's disease Hurler's syndrome Mucopolysaccharidoses (MPS) ... Adults and children over 6 months of age who need regular ...

Mixed tension migraine

... Mixed tension migraine Alternative Names: Headache - mixed tension migraine Treatment : ... Mixed Tension Migraine MLD (Metachromatic leukodystrophy ) MMK (Surgeries for Female Stress Incontinence ) MMR - Vaccine Molar Pregnancy ( Hydatidiform Mole ) ... Migraine without aura (no ...

Molar pregnancy

... : Can a mom's depression affect her baby? Postpartum thyroiditis Pre- ejaculation fluid: Can it cause pregnancy ? MLD (Metachromatic leukodystrophy ) Molar Pregnancy ( Hydatidiform Mole ) Mole ( Birthmarks - Pigmented) Molluscum Contagiosum Mongolian Blue Spots ... age younger than 20 ...

Molluscum contagiosum

... vulgaris, verruca plantar is, HPV, acrochordon, molluscum contagiosum , seborrheic keratosis , and verruca plana; ... MLD (Metachromatic leukodystrophy ) Molar Pregnancy ( Hydatidiform Mole ) Mole ( Birthmarks - Pigmented) Molluscum Contagiosum Mongolian Blue Spots ... CRYOTHERAPY: Using ...

Leukodystrophy in Biological Technology

Shire's New Product Portfolio Delivers Strong Quarterly Performance

... 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.

... 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

... 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 ....