by Madaline Spencer | Feb 14, 2024
For many years, there has been a pressing need for effective treatment options for individuals living with Angelman syndrome. However, the recent Priority Medicine (PRIME) designation by the European Medicines Agency (EMA) for GTX-102 for the treatment of Angelman...
by Madaline Spencer | Feb 12, 2024
Orlando Agrippa, Founder and CEO of Sanius Health, and Oliver Shastri, team lead for sickle cell disease at Pfizer, discuss how social media can foster community for sickle cell disease patients. Sickle cell disease is a disorder in which red blood cells...
by Madaline Spencer | Feb 3, 2024
Krabbe Disease is a rare neurodegenerative disorder caused by mutations in the galactocerebrosidase (GALC) gene. Advancements in Krabbe newborn screening are making early diagnosis possible. Image Source: Unsplash Krabbe disease affects approximately...
by Madaline Spencer | Feb 3, 2024
Ponni Subbiah, MD, MPH, Senior Vice President Global Head of Medical Affairs and Chief Medical Officer, Acadia Pharmaceuticals, discusses trofinetide for the treatment of Rett syndrome. Overview of Rett Syndrome Rett syndrome is a neurodevelopmental condition...
by Madaline Spencer and Peter Ciszewski | Jul 27, 2023
Courtney Silverthorn, Ph.D., Associate Vice President for Science Partnerships at the Foundation for the National Institutes of Health (FNIH), discusses the recent announcement that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has...
