Patisiran was associated with a moderate improvement in cardiac symptoms in patients with transthyretin amyloid cardiomyopathy (ATTR-CM), a study found.

Tafamidis is linked to reduced all-cause mortality and a lower incidence of congestive heart failure exacerbations in ATTR-CM, a study found.

Using the new method, the researchers were able to successfully identify 15 cases of transthyretin mediated amyloid cardiomyopathy (ATTR-CM).

A multimodal approach combining vosoritide and limb lengthening led to gains in stature, gait, and emotional well-being in achondroplasia.

Conducting a RCT of the Accessercise app among adults with physical disabilities, including achondroplasia, is feasible and acceptable.

The change in extracellular volume is the only predictor of adverse outcome in patients with ATTR-CM treated with tafamidis, a study found.

Age over 75 years was significantly associated with reduced survival and shorter time on treatment. Ibrutinib remains an effective therapy for chronic lymphocytic leukemia (CLL), but older patients ...

Therapies for Gaucher disease generally fall into 2 main categories: enzyme replacement therapy (ERT) and substrate reduction therapy (SRT). Neither ERT nor SRT effectively treats the neurological ...

Meta-analysis confirmed the clinical benefits of adeno-associated virus (AAV)-based liver-directed gene therapy for patients with hemophilia.

Senior correspondent Larry Luxner interviews Chris Lewis, whose father, comedian Jerry Lewis, was a tireless advocate for people with muscular dystrophy.

Novel findings in the pathophysiology of HD could unlock a new imaging-based marker for disease burden and clinical decline, a study found.

The CSHA Clinical Frailty Scale is a feasible tool for predicting treatment-related toxicity and discontinuation in older adults with CLL.