Novartis to pursue SMA drug branaplam in Huntington's disease

Novartis is seeking to repurpose its investigational oral spinal muscular atrophy (SMA) drug branaplam to treat Huntington's disease, the Swiss drugmaker said on Wednesday, as it plans a clinical ...
New Scientist

Therapy may be the most effective way to ease irritable bowel syndrome

People with irritable bowel syndrome are often only given treatments like cognitive behavioural therapy after others have ...

After rare diagnosis, Coeur d'Alene girl beating the odds

Doctors told the Coeur d'Alene girl's parents that no research on the condition had been conducted in 20 years.
Coeur d'Alene Press

After rare diagnosis, Cd’A girl beating the odds

COEUR d’ALENE — In her three years of life, Grace Sayler has defied the odds. She received what appeared to be a hopeless ...
Los Angeles Times

Nephrotic Syndrome: Diagnosis, Pathophysiology & Treatment Insights

Nephrotic syndrome is a clinical entity defined by heavy proteinuria, hypoalbuminemia, hyperlipidemia, and edema, stemming from various glomerular diseases. Diagnosis requires a comprehensive ...
Medical Xpress

FDA grants accelerated approval for first treatment for Barth syndrome

The U.S. Food and Drug Administration has granted accelerated approval to Forzinity (elamipretide) injection as the first treatment for Barth syndrome. Barth syndrome is a rare, serious, and ...
HUB

FDA approves drug for treatment of rare mitochondrial disorder

Walker Burger always loved baseball, but attending the games of his favorite team, the Atlanta Braves, used to be a physical challenge. In fact, every form of exercise was difficult. Burger, 35, has a ...
Becker's Hospital Review

FDA approves 1st Barth syndrome treatment

The FDA has granted accelerated approval for the elamipretide injection, the first treatment for Barth syndrome in patients weighing at least 30 kg. Barth syndrome is a life threatening disease of the ...
ABC 7 Chicago

Orland Park baby's treatment for rare genetic disease granted accelerated approval by FDA

ORLAND PARK, Ill. (WLS) -- A baby in the Chicago area is battling a rare disease and desperately needs a drug treatment for a better life. The drug has now been granted accelerated approval by the FDA ...
STAT

FDA approves ultra-rare disease drug that became a symbol of uncertainty over regulatory standards

Ed Silverman, a senior writer and Pharmalot columnist at STAT, has been covering the pharmaceutical industry for nearly three decades. He is also the author of the morning Pharmalittle newsletter and ...
Medical Xpress

Precision genetic target provides hope for Barth syndrome treatment

Researchers at The Hospital for Sick Children (SickKids) have uncovered a promising new therapeutic target for Barth syndrome, a rare genetic condition with no current cure. Barth syndrome is an ...
AOL

Tips and treatments for restless leg syndrome

Dear Savvy Senior, What can you tell me about restless leg syndrome? Over the past year or so I’ve developed an uncontrollable urge to move my legs because of an annoying tingling sensation, and it’s ...