Intrathecal onasemnogene abeparvovec aims to address the genetic root cause of SMA by replacing the SMN1 gene in a single dose.

After starting in clinical medicine at her parents' urging, Dr. Vincent switched to her field of interest: basic research.

Orthopedic management plays a central role in the care of patients with achondroplasia.

TK2d has long existed in a therapeutic void, with patients having few, if any, disease-modifying treatment options.

Lyza Weisman spoke at the recent MDA Clinical & Scientific Conference of no longer being able to do the 'little things' that make a life.

An elderly patient with a history of pulmonary sarcoidosis was diagnosed with transthyretin amyloid cardiomyopathy.

To better achieve clinical progress, therapeutic goals in myasthenia gravis must be objectively and unambiguously defined.

Deciding when and how to treat chronic lymphocytic leukemia (CLL) remains challenging, with MRD-guided strategies offering new insights.

Studies show that ANCA seropositivity can be immensely useful in helping physicians identify a likely final diagnosis.

Healthcare professionals generally agree that genetic testing can be useful in the HAE diagnostic process, but many urge caution in interpreting results.