FDA Grants MDA Kickstart Program Rare Pediatric Disease Designation and Orphan Drug Designation in Congenital Myasthenic Syndrome

Published Date: 22 Dec 2024

The program assembles a collaborative team of in-house and external experts in research, clinical care, and regulatory affairs to navigate gene therapy programs through the drug development process.

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Real-world lecanemab findings show that APOE epsilon 4 status identifies patients at a higher risk for ARIA but cannot predict which individuals will develop the complication.

The FDA has approved a radioactive agent for use ⁠in brain scans to help assess patients for Alzheimer's disease.

Patients who undergo anterior cruciate ligament reconstruction may show persistent neuromuscular alterations during functional tasks, despite reduced joint position sense errors.

Alzheimer disease and LATE-NC pathology were associated with more timely dementia diagnoses, a study shows.

Limiting sugar during pregnancy and infancy is associated with reduced risk for dementia during adulthood, delayed disease onset, and benefits for brain health, a study shows.

Ponesimod is well tolerated and effective for up to 13 years in patients with relapsing-remitting multiple sclerosis, a combined analysis shows.

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