Hypersensitivity reactions in a mouse model prompted the agency to suspend Denali’s planned Phase I development for DNL952 ...

Getting a diagnosis of Pompe disease can be tricky. That's partly because the disorder is so rare. It affects only about 1 in every 40,000 people in the United States. “It’s really not on most ...

Pompe disease is a rare metabolic disorder. It’s marked by a deficiency in lysosomal alpha-glucosidase. Your body needs this enzyme to break down stored glucose, which is called glycogen. When your ...

Doctors can’t cure Pompe disease. But treatment can help babies, children, and adults with the condition live longer, and often with fewer complications. In 2006, the FDA approved the first treatment ...

Pompe disease, a rare genetic disorder that causes severe and often life-threatening heart and lung problems in newborns and young children — and which can also occur later in life, causing ...

Genetic disorders are complex in nature. They are usually rare diseases that occur to person from birth, some of them can even cause life-threatening complications whereas others just form a deformity ...

In the realm of rare diseases, Sanofi has added another arrow to its Pompe quiver. The FDA cleared Sanofi’s Nexviazyme, also known as avalglucosidase alfa-ngpt, to treat patients ages one and older ...

Astellas’ gene therapy ambitions have faced yet another setback. The FDA has slammed the brakes on a phase 1/2 study of the Japanese pharma’s Pompe disease med while the regulator investigates a ...

Rare genetic disorder affects 1 in 40,000 people. — -- President Donald Trump's first address to a joint session of Congress happened to fall on Rare Disease Day -- a fact he highlighted by ...

Megan Crowley attends a joint session of the U.S. Congress. (Photo by Win McNamee/Getty Images) During his joint address to Congress tonight, Feb. 28, President Donald Trump called special attention ...

As Sanofi's Pompe disease franchise fuels its growth in rare diseases, the company has inked a $180 million settlement related to a license for a key medicine. Sanofi Genzyme has agreed to shell out ...

Pompe disease is a rare genetic condition that affects only 1 person in every 40,000 people in the United States, according to the National Institute of Neurological Disorders and Stroke (NINDS).