“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....
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...
Our Executive Director Mr. Prasanna Shirol was a part of First Ever summit dedicated to advancing equitable care for rare diseases in older adults. The meeting was attended from 25 countries across Canada, Latin America, Europe, and Asia, representing patient and caregiver communities, patient advocates, healthcare professionals, and healthcare policy experts. This is a short video highlighting the impact of the initiative to date, in the words of the COLLABORATE Community.
