Pompe disease is a rare, genetic disorder that results from the deficiency of the enzyme acid alpha-glucosidase (GAA). This results in the accumulation of glycogen in the muscles and organs, leading ...

Reflecting on my journey with Pompe disease, I’m reminded of the countless times I’ve been asked, “But you look fine, what’s wrong?” The truth is, that Pompe is an invisible condition, hiding in plain ...

After a decade of searching for an answer to what was causing progressive pain and weakness in my body, I was finally diagnosed with Pompe disease. Pompe, a rare genetic disorder, has long presented a ...

Sanofi has added another string to its Pompe disease therapy bow, after getting FDA approval for Nexviazyme, its latest therapy for the rare, inherited disorder. The US regulator has approved ...

Ian MacPherson has had to overcome more challenges in his lifetime than any person should ever have to endure. The Morpeth man has one more obstacle he’d like to clear – to get a job.

Bengaluru: The medical education department is reaching out to corporate entities for support in treating children affected ...

At a time when America is facing unprecedented debt, with inflation hitting hardworking families and entitlement programs ...

and Pompe disease. In January, Dyne Therapeutics revealed new data from its ongoing Phase 1/2 ACHIEVE trial of DYNE-101, the company’s lead candidate, in patients with myotonic dystrophy type 1 ...

Parkinson's disease is an illness that affects the part of your brain that controls how you move your body. Some people with Parkinson’s first notice weakness, difficulty walking, and stiff muscles.

Rome in 65 AD, the philosopher Seneca has been Emperor Nero‘s teacher, mentor and close advisor since childhood, he is significantly involved in his ascent. Nevertheless, Nero gets weary of Seneca and ...

There are a lot of ways to assess a movie theater. The beauty of the architecture and its historical significance certainly count for a large part. But those aren’t the only factors. To find ...