Genethon Signs Licensing Agreement With AskBio for the Development of an Investigational Gene Therapy for Pompe Disease

Genethon, a leading laboratory in gene therapy for rare diseases, today announced that it has entered into an exclusive, worldwide licensing agreement with AskBio, a gene therapy company wholly owned ...
MedCity News

Sanofi Abandons Deal for Rare Disease Drug Amid FTC’s Monopoly Concerns

Sanofi is walking away from a deal to license an experimental Maze Therapeutics drug for a rare enzyme deficiency after the Federal Trade Commission challenged the transaction as anti-competitive. The ...

Belief BioMed Congratulates Partner AskBio on IND Acceptance by FDA for Investigational Gene Therapy for Late-Onset Pompe Disease

AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, recently announced that the United States Food and Drug Administration (FDA) has ...
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 ...

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, ...
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 ...
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 ...
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 ...
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 ...
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 ...
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 ...