Amicus Therapeutics FOLD reported adjusted earnings of 17 cents per share for the third quarter of 2025, which beat the Zacks ...

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 ...

Q3 2025 Management View Bradley Campbell, CEO, highlighted another quarter of double-digit revenue growth in the Fabry and ...

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, 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 ...

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 ...

Roche has punted a Pompe disease gene therapy from its pipeline after weighing up the competitive landscape. The Swiss drugmaker made the change in a second-quarter update that also featured news of ...

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 ...

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 ...

In early 2022, Connor started a new medication called Nexviazyme, but each treatment took 13 hours. This summer, he received ...

If you or your child has Pompe disease, it's important to know that each case is different, and that doctors have treatments to help manage it. Pompe disease happens when your body can't make a ...