Contributed by Dravet Foundation Spain The group values the progress made but urges the continued implementation of the effectiveness of the drugs Epilepsy unexpectedly and suddenly marks the onset of…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
Blood transfusions are a vital medical procedure used to address a multitude of health conditions and emergencies, such as severe anemia, sepsis, trauma, internal bleeding, hemophilia, sickle cell disease, thalassemia,…
The American Society of Gene and Cell Therapy (ASGCT) held its 27th Annual Meeting from May 7-11, 2024. During the conference, stakeholders in the field of cell therapy come together…
Have you heard of antisense oligonucleotides (ASOs)? These short, synthetic strands of nucleic acids are designed to bind specifically to the mRNA transcripts of genes, blocking the production of disease-causing…
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