Posts by: Joana Carvalho

Joana Carvalho

July 3, 2019July 3, 2019

Amicus and Paragon Working to Advance Potential Gene and Other Therapies for Rare Diseases Like Pompe

News

Amicus Therapeutics announced that it has entered into a manufacturing agreement with Paragon Bioservices to drive the development of technologies, including gene therapies, now in preclinical testing for the treatment of ... Read more

May 22, 2019May 22, 2019

New Genetic Variant May Help Explain Variability of Symptom Onset, Study Says

News

Scientists have discovered a genetic variant that might help explain why some people with Pompe disease experience their first symptoms earlier or later in life. The study, “A genetic ... Read more

March 13, 2019March 13, 2019

Rare Childhood Disorders Pose Significant Impact on Quality of Life of Children and Parents, Study Reports

News

Rare childhood disorders, including Pompe disease, pose a significant estimated impact on the quality of life of affected children and their parents, a study reports. The study, “Health utilities and ... Read more