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....
June 28 is International Newborn Screening Day. The purpose of newborn screening is to identify conditions which can be treated, especially if caught early, so that affected children can live...
KEM Hospital, one of the 11 Centres of Excellence (CoE) for Rare Diseases in India, is facing challenges in fulfilling its mandate due to a lack of designated space and...
