Patisiran Promising for Transthyretin-Mediated Amyloidosis With Cardiomyopathy
The APOLLO-B study included patients 18 years of age and older with ATTR amyloidosis (hereditary or wild-type) with cardiomyopathy.
Diagnosis & Disease Information
Eplontersen Bests Placebo in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy Study
Eplontersen is an investigational ligand-conjugated antisense medicine designed to reduce the production of TTR protein to treat both hereditary and nonhereditary forms of ATTR.
Imcivree Approved for Weight Management in Bardet-Biedl Syndrome
Imcivree is a melanocortin 4 receptor (MC4R) agonist that re-establishes MC4 receptor pathway activity to reduce hunger and promote weight loss.
Vutrisiran to Undergo FDA Review for Polyneuropathy of Hereditary ATTR Amyloidosis
Vutrisiran is an investigational, subcutaneously-administered RNAi therapeutic designed to block the production of wild-type and mutant transthyretin protein.
Omaveloxolone Gets Priority Review for Friedreich Ataxia
Omaveloxolone is an investigational Nrf2 activator.
Cuvrior Gets FDA Approval for Wilson Disease
The approval was based on data from a phase 3 study that compared Cuvrior with penicillamine in 53 adults with Wilson disease.
AMPK Activator Gets Fast Track Status for X-Linked Adrenoleukodystrophy
PXL770 is a novel, first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator.
Psychotherapy May Help Sleep Apnea, Obesity, and NAFLD
Nonalcoholic fatty liver disease often has several systemic comorbities, including obstructive sleep apnea. Researchers explored a psychological link between them.
FDA Requests Clinical Outcomes Data for Galactosemia Treatment
Galactosemia is a rare genetic metabolic disease caused by an inability to metabolize galactose.
Shift Work Disorder: When and How to Shift Workers’ Gears
Approximately 5% to 10% of shift workers, including many health care professionals, suffer from shift work disorder, a circadian sleep-wake cycle disorder defined by poor quality sleep.
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