Posts by: Alice Melao

News

Amicus Therapeutics is planning to launch a pivotal trial to compare its investigational therapy AT-GAA (ATB200/AT221) to the current standard of care available for patients with Pompe disease. The new ... Read more

July 25, 2018July 25, 2018

Quantitative Muscle MRI Can Help Monitor Late-onset Pompe Disease, Study Shows

News

Magnetic resonance imaging (MRI) seems to be an effective and efficient tool to evaluate muscular status and monitor patients with late-onset Pompe disease (LOPD), according to researchers. Their study, ... Read more

July 18, 2018July 18, 2018

Higher Lumizyme Dose May Help Prevent Pompe Progression in Infants, Case Report Suggests

News

Treatment with Lumizyme (alglucosidase alfa) at double the approved dose may help prevent the rapid decline of respiratory and cardiovascular functions in infants with Pompe disease, a case report suggests. ... Read more

June 14, 2018June 14, 2018

Infantile-onset Pompe Disease Cases Highlight Importance of Early Diagnosis, Treatment

News

Four cases of infantile-onset Pompe disease recently reported in Australia help shed light on some of the unique challenges care providers face in diagnosing and managing this genetic disease. ... Read more

May 23, 2018May 23, 2018

NIH Awards $325K Small Business Grant to Develop Risk Assessment Tool for Pompe Disease

News

The National Institutes of Health (NIH) has awarded EpiVax with a Small Business Innovation Research (SBIR) grant of $324,980. The company is going to use the funding to further ... Read more

May 16, 2018May 16, 2018

ERT Fails to Prevent Long-Term Effects of Infantile Pompe Disease to Brain, Study Shows

News

Treatment with enzyme replacement therapy can help children with classic infantile Pompe disease reach adulthood. However, it fails to prevent the long-term effects of the disease in the brain and ... Read more

May 9, 2018May 9, 2018

Amicus Recruiting Pompe Patients for Phase 1/2 Trial of AT-GAA Combo Therapy

News

Amicus Therapeutics is recruiting participants for the fourth group of its ongoing Phase 1/2 clinical trial evaluating the company’s investigative combination therapy AT-GAA. The trial (NCT02675465), called ATB200-02, will ... Read more

April 11, 2018April 11, 2018

Duke Researchers Initiating Phase 1 Trial on New Gene Therapy for Pompe Disease

News

Duke Health researchers are planning to launch a Phase 1 trial to test the safety of a new investigational gene therapy they developed to treat patients with Pompe disease. ... Read more

Posts by: Alice Melao

Alice Melao

Amicus Announces Plans for Developing AT-GAA to Treat Pompe Disease

Quantitative Muscle MRI Can Help Monitor Late-onset Pompe Disease, Study Shows

Higher Lumizyme Dose May Help Prevent Pompe Progression in Infants, Case Report Suggests

Infantile-onset Pompe Disease Cases Highlight Importance of Early Diagnosis, Treatment

NIH Awards $325K Small Business Grant to Develop Risk Assessment Tool for Pompe Disease

ERT Fails to Prevent Long-Term Effects of Infantile Pompe Disease to Brain, Study Shows

Amicus Recruiting Pompe Patients for Phase 1/2 Trial of AT-GAA Combo Therapy

Duke Researchers Initiating Phase 1 Trial on New Gene Therapy for Pompe Disease

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