Pompe disease, a lysosomal storage disorder, is characterized by an impairment of glycogen degradation that is caused by a deficiency of the enzyme acid α-glucosidase (GAA).1 In efforts to expand the...
“I watch my 16-month-old daughter Sera bravely fight an ultra-rare disease every day,” said a tearful Michael Andrew, a desperate father waging a war not against the world but against...
Alexion, AstraZeneca Rare Disease, has completed a definitive purchase and license agreement for a portfolio of preclinical rare disease gene therapy programes and enabling technologies from Pfizer Inc. (Pfizer). These...
Three new mutations in the GAA gene were linked with heart disease and severe breathing problems in infants with infantile-onset Pompe disease (IOPD) in Malaysia, a study reports. “The novel mutations identified in this...
Gene therapy work was started 10 years ago under the guidance of founder and former chairman Dr K Bhujang Shetty. BENGALURU: Gene therapy human trials will be initiated in India for...
Boehringer Ingelheim Our team from the India, Middle East, Turkey, and Africa region recently organized a rare disease summit, bringing together healthcare professionals, patient organization representatives, and policy makers....
