Between 2001 to 2011, annual age-adjusted incidence increased from 0.55/100,000 to 0.78/100,000, demonstrating an increasing trend with age. Incidence peaks in individuals over 70 years of age ...

The modified compounds achieved up to a 3.01-fold enhancement in GCase activity and 11.26-fold increase in GAA activity. Specifically designed and synthesized neutral thiourea-type sp 2-iminosugars ...

After 1 year, participants with normal sleep patterns showed superior quality of life, fewer behavioral problems, less mental fatigue, and more physical activity. Patients with Huntington disease (HD) ...

A subgroup of patients with HD with global memory impairments, particularly consolidation deficits, showed more medial temporal lobe atrophy. Patients with Huntington disease (HD) show deficits in ...

Generalized pustular psoriasis (GPP) is a rare, severe form of psoriasis characterized by the acute onset of eruptions of sterile pus-filled skin lesions and rashes with or without systemic ...

The majority (79%) of cardiac amyloidosis cases were responsible for sudden cardiac death. Cardiac amyloidosis can be associated with sudden cardiac death and is often undiagnosed during a person’s ...

Inebilizumab-cdon (Uplizna ®) has received US Food and Drug Administration (FDA) approval for the treatment of adults with generalized myasthenia gravis (MG) with antibodies against the acetylcholine ...

There was a reduction in volume of involvement, cardiac pyrophosphate activity, native T1, and extracellular volume during a median of 369 days between scans in patients with ATTR-CM being treated.

Patients diagnosed with wild-type ATTR-CM on or before 65 years of age had better survival after diagnosis compared to patients who were diagnosed after 65 years. Wild-type transthyretin-mediated ...