Lysosomal Storage Disorders News and Research

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BioMarin Pharmaceutical acquires ZyStor Therapeutics, furthers development of ZC-701 for Pompe disease

BioMarin Pharmaceutical acquires ZyStor Therapeutics, furthers development of ZC-701 for Pompe disease

BioMarin Pharmaceutical Inc. announced today that it has acquired ZyStor Therapeutics, Inc. (ZyStor), a privately-held biotechnology company developing enzyme replacement therapies (ERT) for the treatment of lysosomal storage disorders. ZyStor's lead product candidate is ZC-701, a novel fusion of insulin-like growth factor 2 and alpha glucosidase (IGF2-GAA) in development for Pompe disease.

18 Aug 2010

ADDF awards $210,300 to Amicus Therapeutics to evaluate PCs for treating Alzheimer's disease

ADDF awards $210,300 to Amicus Therapeutics to evaluate PCs for treating Alzheimer's disease

The Alzheimer's Drug Discovery Foundation announced today that it has awarded a grant of $210,300 to Amicus Therapeutics (Cranbury, NJ) to evaluate small-molecule, orally delivered pharmacological chaperone compounds as a novel approach for treating Alzheimer's disease.

7 May 2010

Gene therapy may help correct molecular flaws that cause brain disease

Gene therapy may help correct molecular flaws that cause brain disease

Neuroscientists have forged an unlikely molecular union as part of their fight against diseases of the brain and nervous system.

9 Jan 2010

New technique to treat metabolic disorders in babies while still in the womb

New technique to treat metabolic disorders in babies while still in the womb

Researchers say a new development in cord blood transplants for inherited metabolic disorders may be curative for some babies who are treated while still in the womb.

20 Oct 2009

Amicus Therapeutics presents positive results from phase 2 extension study of Amigal for Fabry disease

Amicus Therapeutics presents positive results from phase 2 extension study of Amigal for Fabry disease

Amicus Therapeutics has announced positive results from its ongoing Phase 2 extension study of its investigational drug, Amigal (migalastat HCL) for Fabry disease.

29 Mar 2009

Fruit flies provide new insights into a brain-damaging disease mucolipidosis type IV

Fruit flies provide new insights into a brain-damaging disease mucolipidosis type IV

Johns Hopkins researchers have used fruit flies to gain new insights into a brain-damaging disorder afflicting children.

26 Nov 2008

Genzyme receives Japanese marketing approval for Elaprase

Genzyme receives Japanese marketing approval for Elaprase

Genzyme Corp. has announced that it has received approval to market Elaprase (idursulfase) in Japan for the treatment of Hunter syndrome.

4 Oct 2007

Novel preclinical gene therapy approach for treating Niemann-Pick disease

Novel preclinical gene therapy approach for treating Niemann-Pick disease

Genzyme Corp. has announced the publication of a new study in The Proceedings of the National Academy of Sciences describing a novel preclinical gene therapy approach for treating Niemann-Pick disease.

24 May 2007

New drug to treat rare Pompe disease gets FDA approval

New drug to treat rare Pompe disease gets FDA approval

The Food and Drug Administration in the U.S. has given approval for the first drug to treat a rare, debilitating muscle disease that can kill infants within a year.

1 May 2006

Genzyme begins gene transfer trial for peripheral arterial disease

Genzyme begins gene transfer trial for peripheral arterial disease

Genzyme Corporation announced today that the first patient has been treated in a new phase 2 clinical trial examining the safety and effectiveness of locally delivered gene transfer for patients with peripheral arterial disease.

24 Feb 2005

Genzyme files for European approval of Myozyme for treatment of Pompe disease

Genzyme files for European approval of Myozyme for treatment of Pompe disease

Genzyme Corp. announced today that the European Medicines Agency (EMEA) has accepted its marketing authorization application for Myozyme(R) (alglucosidase alfa), an investigational enzyme replacement therapy for Pompe disease.

20 Dec 2004

Disruption of protein folding causes neurodegeneration, mental retardation

Disruption of protein folding causes neurodegeneration, mental retardation

Excess accumulation in brain cells of a fat molecule called GM1-ganglioside (GM1) disrupts the folding of newly assembled proteins into their proper shapes, triggering nerve degeneration and mental retardation in children.

10 Sep 2004

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