Seeking children aged 6 months and older with Tay-Sachs or Sandhoff disease for a study of the safety of an experimental gene therapy and whether it improves their ability to move.
5 Studies found
This study is intended to collect data from medical records on patients who have Duchenne Muscular Dystrophy
Seeking Male and Female age 0-25 years who may have cancer or another condition that is characterized by abnormal cell growth but may be benign (not cancer), or may already have been diagnosed with cancer.
This study is collecting tumor tissue, body fluids and blood samples from patients undergoing biopsies or surgery to gain a better understanding of different cancers.
This is a registry of Pyruvate Kinase patients to study how the disease affects different people and how it is treated around the world.