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Gene Therapy Shows Long-Term Vision Benefits in Rare Eye Disease

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

Dec 22, 2024
A computer rendering of a transparent head with the eye and optic nerve highlighted.
First Drug Approved for Familial Chylomicronemia Syndrome

Antisense oligonucleotide olezarsen slashes high triglycerides

Dec 20, 2024
FDA APPROVED olezarsen (Tryngolza) over a computer rendering of fats in the bloodstream.
FDA Approves First Drug for Classic Congenital Adrenal Hyperplasia in 70 Years

Crinecerfont reduces excessive adrenal androgen production, lowering glucocorticoid dose

Dec 16, 2024
FDA APPROVED crinecerfont (Crenessity) over a computer rendering of a transparent body with the adrenal glands highlighted.
Racial Diversity Lacking in Epilepsy Genetics Studies

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

Dec 09, 2024
AES over a photo of Los Angeles Convention Center
Beta-Blockers Tied to Delayed Huntington's Disease Progression

Symptom onset was later; rate of worsening was slower

Dec 02, 2024
A computer rendering of neuronal inclusions in Huntington’s disease.
FDA Investigating Blood Cancer Risk With Gene Therapy Skysona

Agency is evaluating the need for further regulatory action

Nov 27, 2024
A photo of a woman holding her boy who suffers from adrenoleukodystrophy.
Medications to Reduce Lipoprotein(a); Genome Models and CVD Risk

Also in TTHealthWatch: genetic testing of couples planning pregnancy

Nov 23, 2024
TTUHSC EL PASO HEALTHWATCH with their logo and photos of Rick Lange, MD, and Elizabeth Tracey
Fitness Level May Offset Genetic Dementia Risk

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

Nov 19, 2024
A photo of a woman riding a stationary bicycle in a gym.
Gene Therapy Promising for Rare Genetic Cardiomyopathy

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

Nov 18, 2024
AHA over a photo of McCormick Place Convention Center in Chicago, IL.
Oral Drug Safe, Effective in Kids With Achondroplasia

No adverse events resulted in treatment discontinuation

Nov 18, 2024
A photo of a group of boys affected by achondroplasia.
First Brain-Delivered Gene Therapy Approved for AADC Deficiency

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

Nov 14, 2024
FDA APPROVED eladocagene exuparvovec-tneq (Kebilidi) over a computer rendering of nerve cells.
Lowest Paid Medical 鶹ý in 2024

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

Nov 14, 2024
 The YouTube thumbnail with a cartoon man in scrubs holding a piggy bank next to the number $244,517.
Kidney Disease Genetic Risk Common in West Africans

Observational study points to genetic risk allele dose-response

Oct 29, 2024
ASN over a photo of San Diego Convention Center in San Diego, CA.
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