Search results for "rare disease"

Repeated Liver Tests for Elevated Enzymes Reveal Unexpected Cause: Muscle Inflammation

Repeated Liver Tests for Elevated Enzymes Reveal Unexpected Cause: Muscle Inflammation

A 50-year-old woman surnamed Kim sought medical care after experiencing persistent fatigue and difficulty climbing stairs or lifting heavy objects.
FDA Approves Emcitate as First Treatment for Rare Genetic Disorder That Starves Brain of Thyroid Hormone

FDA Approves Emcitate as First Treatment for Rare Genetic Disorder That Starves Brain of Thyroid Hormone

Thyroid hormone is essential for infant brain development. But when the cellular "gateway" that transports the hormone into the brain is defective...
“I Dream of an Ordinary Life”: Expanded Insurance Coverage Brings New Hope to Dravet Syndrome Families

“I Dream of an Ordinary Life”: Expanded Insurance Coverage Brings New Hope to Dravet Syndrome Families

“Until just before my daughter turned 2, she frequently suffered not only febrile seizures but also afebrile seizures.
Hanmi Completes Key Phase 2 Dosing for Once-Weekly Rare Disease Drug Efpeglucagon

Hanmi Completes Key Phase 2 Dosing for Once-Weekly Rare Disease Drug Efpeglucagon

During normal digestion, dietary carbohydrates convert into glucose that circulates in the blood.
Deciphering Ampullary Cancer: Korean Researchers Identify Aggressive Subtype of Rare Disease

Deciphering Ampullary Cancer: Korean Researchers Identify Aggressive Subtype of Rare Disease

Where the bile duct and pancreatic duct meet to drain digestive juices into the duodenum, a rare and elusive malignancy...
550 Days in Limbo: Groups Demand 100-Day Fast-Track Insurance Coverage for Breakthrough PAH Drug Winrevair

550 Days in Limbo: Groups Demand 100-Day Fast-Track Insurance Coverage for Breakthrough PAH Drug Winrevair

With national health insurance coverage for the new pulmonary arterial hypertension treatment Winrevair (active...
Biogen Korea Taps Pharma Veteran Kim Chul-woong as New Country Lead

Biogen Korea Taps Pharma Veteran Kim Chul-woong as New Country Lead

Biogen Korea has appointed Kim Chul-woong as its new general manager, tapping an executive with extensive expertise in...
Expanded Health Insurance Coverage Lowers Barrier to Care for Rare Heart and Kidney Disorder

Expanded Health Insurance Coverage Lowers Barrier to Care for Rare Heart and Kidney Disorder

Patients suffering from light-chain amyloidosis, a rare and debilitating blood disorder that causes abnormal proteins...
Predicting a Rare Disease from the Back of Your Hand: AI Breakthrough in Acromegaly Detection

Predicting a Rare Disease from the Back of Your Hand: AI Breakthrough in Acromegaly Detection

While palm lines are traditionally used in folklore to predict the future, a new scientific study suggests that the...
Decoding the Blueprint of Aging: Scientists Map 7 Million Cells Across 21 Organs

Decoding the Blueprint of Aging: Scientists Map 7 Million Cells Across 21 Organs

We often attribute the appearance of wrinkles, declining stamina, and increased disease risk simply to "getting older.