Following Sanofi’s decision to terminate its proposed acquisition of an exclusive license to Maze Therapeutics Inc.’s developmental drug to treat Pompe disease, the Federal Trade Commission moved to dismiss its...
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...
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.
