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...
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....
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...
Hereditary Angioedema (HAE) is a rare genetic condition that can significantly impact a patient’s quality of life. The journey of living with HAE is often marked by uncertainty, frustration, and...
By Monika Yadav Express News Service Sanofi Specialty Care, which has a portfolio of drugs for rare diseases, has received a recommendation from the Subject Expert Committee of the Drugs Controller...
By Daphne Clarance: In February, the death of former Pakistan president Pervez Musharraf due to amyloidosis made headlines across the globe. As per reports, Google searches peaked with this rare...
