Pompe Disease News and Research

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Pompe disease is a rare and often fatal muscle disease caused by an inherited deficiency of the enzyme acid alpha-glucosidase, which is responsible for breaking down glycogen within cells. Pompe disease ranges from a rapidly fatal infantile-onset form with severe cardiac involvement to a more slowly progressive late-onset form primarily affecting skeletal muscle. There is currently no therapeutic treatment available for the disease, which affects an estimated 5,000-10,000 people worldwide.
UF administers Lumizyme for late-onset Pompe disease

UF administers Lumizyme for late-onset Pompe disease

UF doctors administer newly available therapy for late-onset Pompe disease

UF doctors administer newly available therapy for late-onset Pompe disease

Lumizyme receives FDA approval for patients with late-onset Pompe disease

Lumizyme receives FDA approval for patients with late-onset Pompe disease

Genzyme's Lumizyme for Pompe disease receives FDA marketing approval

Genzyme's Lumizyme for Pompe disease receives FDA marketing approval

Genzyme signs consent decree of permanent injunction, agrees to disgorgement

Genzyme signs consent decree of permanent injunction, agrees to disgorgement

Genzyme to initiate $2 billion stock buyback

Genzyme to initiate $2 billion stock buyback

LOTS results of Myozyme in older children and adults with Pompe disease published

LOTS results of Myozyme in older children and adults with Pompe disease published

Enobia Pharma unveils findings from first HPP self-reported patient survey at ACMG 2010

Enobia Pharma unveils findings from first HPP self-reported patient survey at ACMG 2010

Pathway Genomics' personal Genetic Health Report now available

Pathway Genomics' personal Genetic Health Report now available

Genetic Disorder Tracker introduced by Guidepoint Global

Genetic Disorder Tracker introduced by Guidepoint Global

Genzyme announces fourth-quarter and full-year 2009 financial results

Genzyme announces fourth-quarter and full-year 2009 financial results

Special issue reviews current state of science in fetal therapy

Special issue reviews current state of science in fetal therapy

Amicus Therapeutics presents additional data from Amigal Phase 2 extension study for Fabry disease

Amicus Therapeutics presents additional data from Amigal Phase 2 extension study for Fabry disease

Simple genetic test can help in detecting heart abnormalities

Simple genetic test can help in detecting heart abnormalities

UF researchers work out to find new treatments for Pompe disease

UF researchers work out to find new treatments for Pompe disease

Genzyme receives PDUFA date from FDA for its Biologics License Application for Lumizyme

Genzyme receives PDUFA date from FDA for its Biologics License Application for Lumizyme

Amicus Therapeutics presents corporate outlook for 2010 at Annual J.P. Morgan Healthcare Conference

Amicus Therapeutics presents corporate outlook for 2010 at Annual J.P. Morgan Healthcare Conference

Genzyme announces reopening of enrollment in its Pompe disease ATAP program

Genzyme announces reopening of enrollment in its Pompe disease ATAP program

Genzyme receives FDA complete response letter for its Lumizyme marketing application

Genzyme receives FDA complete response letter for its Lumizyme marketing application

Shire announces third quarter 2009 financial results

Shire announces third quarter 2009 financial results