AskBio Announces FDA Acceptance of Investigational New Drug (IND) Application for AB-1009 Gene Therapy for Treatment of Late-Onset Pompe Disease (LOPD)

AB-1009 is an investigational gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated. For more information, ...
Healthline

Pompe Disease and Nutrition: A Starter Guide

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 ...
WebMD

Diagnosing Pompe Disease

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 ...

Once fatal in infancy, Pompe Disease patients now surviving into adulthood, thanks to Duke research

New research from Duke University shows patients diagnosed with infantile-onset Pompe disease are surviving into their 20s ...
FierceBiotech

Roche pumps brakes in Pompe disease, axing gene therapy after assessing competition

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 ...
Everyday Health

Pombiliti Plus Opfolda Approved for Late-Onset Pompe Disease

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 ...
FiercePharma

Sanofi notches another Pompe win with FDA blessing for enzyme replacement therapy Nexviazyme

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 ...
Yahoo

Meet Megan Crowley, the Rare Pompe Disease Survivor Trump Saluted in His Speech

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 ...
Onlymyhealth

Pompe Disease: All You Should Know About This Genetic Disorder

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 ...
The Financial Express

Pompe Disease in India | How poor awareness, misdiagnosis and expensive treatment options are affecting patients of this rare disease

There are ongoing efforts to improve the diagnosis, management, and overall quality of life for individuals with Pompe disease in India. (Image Credit: Freepik) With limited treatment options, rare ...
Hindustan Times

Paige Shiver suffering from rare Pompe disease? All we know about her diagnosis amid Sherrone Moore saga

Sherrone Moore was arrested amid allegations of an inappropriate relationship with staff member Paige Shiver, who has a rare condition called Pompe disease. Former University of Michigan football ...
WebMD

Pompe Disease

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 ...