The L-index may help distinguish pseudohypertriglyceridemia caused by glycerol kinase deficiency from severe ...
GV101, a ROCK2 inhibitor for Friedreich ataxia (FA) that may increase frataxin, received Fast Track designation from the FDA.
Up to 75% of all OB/GYNs experience professional burnout, driven by a complex combination of clinical trauma, legal anxiety, ...
A mom shares why revaccinating her son, who has ALGS, brings up fears and how she works with their care team to overcome her ...
The FDA placed a partial hold on Bright Minds’ planned trial of BMB-101, a 5-HT2C agonist for seizures linked to DEEs, ...
The median age at onset of MuSK-positive MG rose by 11 years from the 1980s to 2013 to 2025 at a tertiary center in Italy.
In adults with SMA, epidural spinal cord stimulation increased hip motion during walking without prompting compensatory ...
The SPPB offers a multidimensional functional assessment with prognostic utility comparable to that of the 6-minute walk test ...
The Pulmonary Fibrosis Foundation and Brainomix have entered into a partnership to support the care of people living with ILD ...
In a recent study, serial 99mTc-PYP SPECT/CT imaging showed a significant drop in myocardial tracer uptake in patients with ...
Columnist Faye Adams wonders if health care providers could do more to ensure ATTR-CM specialized care is more accessible to ...
Acoramidis significantly reduced the decline in heart failure-related health status and health-related quality of life in ...
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