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.

Generalized pustular psoriasis (GPP) contributes to consistently negative outcomes across various quality of life parameters.

To make a real difference in the lives of patients with SCD, there should be a focus on optimizing care across a vast spectrum of concerns.

With the characterization of IgG4-RD, this disease can finally be managed in a comprehensive manner—provided that it is correctly diagnosed, of course. 

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.