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 ...
A new clinical development program, PALM, is testing TPIP in 3 active clinical trials in patients with PPF and other ...
The FDA placed a partial hold on Bright Minds’ planned trial of BMB-101, a 5-HT2C agonist for seizures linked to DEEs, ...
In adults with SMA, epidural spinal cord stimulation increased hip motion during walking without prompting compensatory ...
Columnist Faye Adams wonders if health care providers could do more to ensure ATTR-CM specialized care is more accessible to ...
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.
The SPPB offers a multidimensional functional assessment with prognostic utility comparable to that of the 6-minute walk test ...
In a recent study, serial 99mTc-PYP SPECT/CT imaging showed a significant drop in myocardial tracer uptake in patients with ...
Acoramidis significantly reduced the decline in heart failure-related health status and health-related quality of life in ...
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