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YouTuber Dr. Mike looks at the impact of uncommon genetic conditions

RNAi drug led to modest changes in disability, severity, and quality of life over 5 years

However, FDA approval isn't expected soon due to quirk in earlier results

Antisense oligonucleotide olezarsen slashes high triglycerides

Crinecerfont reduces excessive adrenal androgen production, lowering glucocorticoid dose

But more diverse studies have found more genomic links to seizure conditions

Symptom onset was later; rate of worsening was slower

Agency is evaluating the need for further regulatory action

Also in TTHealthWatch: genetic testing of couples planning pregnancy

In middle-age and older adults, high cardiorespiratory fitness was linked with better cognition

Phase I results show "big improvement" for what is usually progressive hypertrophy, expert says

No adverse events resulted in treatment discontinuation

Novel treatment is indicated for children and adults with the rare disorder

These eight specialties earn the least, with many averaging less than $300k per year