Vanessa Vogel-Farley, Executive Director, Dup15q Alliance discusses her Alliance and some of the research for Dup15q syndrome, the common name for chromosome 15q11.2-q13.1 duplication syndrome.
Acadia Pharmaceuticals and Neuren Pharmaceuticals announced that they have entered into an exclusive North American License Agreement for the development and commercialization of trofinetide for Rett syndrome and other indications.
Mr. Robert (Bob) E. Ward, Chairman of the Board and Chief Executive Officer of Eloxx Pharmaceuticals discusses the role of advocacy groups in drug development, especially for rare diseases such as cystic fibrosis (CF).
Daniel de Boer, Founder and CEO of ProQR, discusses his company’s working relationships with various rare disease advocacy groups.
John Schall is Chief Executive Officer of Caregiver Action Network (CAN) and in this video he discusses the mission of the CAN and the many valuable resources his group offers. The mission of the Caregiver Action Network is to promote resourcefulness and respect for the more than 90 million family …
Mr. Robert (Bob) E. Ward, Chairman of the Board and Chief Executive Officer of Eloxx Pharmaceuticals provides an overview of his company and it’s focus on cystic fibrosis.