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FDA Approves First Treatment for Cerebrotendinous Xanthomatosis, a Rare Lipid Storage Disease

The FDA approved Ctexli (chenodiol) for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Ctexli is the first FDA-approved drug to treat CTX, a very rare lipid storage disease.

Pfizer to Discontinue Its Hemophilia B Gene Therapy

(MedPage Today) -- Pfizer will no longer develop and commercialize its hemophilia B gene therapy fidanacogene elaparvovec (Beqvez) across all global markets, according to multiple reports.

Texas Measles Outbreak Nears 100 Cases, Raising Concerns About Undetected Spread

Health officials expect a measles outbreak in West Texas to exceed 100 cases because of low vaccination rates and undetected infections.

Huge Cuts in NIH Research Funding Go Before a Federal Judge

(MedPage Today) -- BOSTON -- A court battle is set to resume Friday over the Trump administration's drastic cuts in medical research funding that many scientists say will endanger patients and dela

On the Death of a Loved One

(MedPage Today) -- There is a loss of words with the sudden death of a loved one. To say too little is offensive and somehow diminishes your pain.

Every hour children spend on screens raises chance of myopia, study finds

Experts warn young people should have limited use of devices and spend more time outdoorsEvery hour young people spend in front of screens increases their chance of being shortsighted, researchers hav

Crossing State Lines for Cancer Care

(MedPage Today) -- Many older Americans with cancer travel across state lines to receive care, according to an analysis of Medicare claims data.

Lung Cancer Screening Population in Real World May Be at Greater Risk for Harm

(MedPage Today) -- Lung cancer screening populations are older and sicker than those in the National Lung Screening Trial (NLST), a multicenter analysis showed, casting doubt on replicability in the

Here's How Patients With Obesity Can Avoid Joint Replacement Revision

(MedPage Today) -- British patients with obesity undergoing hip and knee replacements had less of a need for subsequent revision when, after surgery, they lost weight by using medications such as GLP

Treating Generalized Myasthenia Gravis

(MedPage Today) -- Recently approved treatments for generalized myasthenia gravis (gMG), including complement and neonatal Fc receptor (FcRn) inhibitors, offer a new treatment paradigm, though challe

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