Glycogen Storage Diseases (GSDs) are a group of inherited metabolic disorders that result from deficiencies in enzymes responsible for the synthesis or breakdown of glycogen, a stored form of glucose.

Polyglucosans are glycogen molecules with overlong chains, which are hyperphosphorylated in the neurodegenerative Lafora disease (LD). Brain polyglucosan bodies (PBs) cause fatal neurodegenerative ...

Ultragenyx Pharmaceutical is a mid-cap biotech firm with a promising pipeline and an optimistic outlook from analysts. Click ...

Beam Therapeutics BEAM is developing its lead pipeline candidate, BEAM-101, a base-editing therapy, in the phase I/II BEACON study for treating sickle cell disease (SCD), an inherited blood disorder.

Increased Glycogen Storage: Creatine may boost glycogen (carbohydrate ... However, those with pre-existing conditions such as ...

Severe metabolic liver disorders and unmet medical needs that are glycogen storage diseases type I (GSDI) and familial hypercholesterolemia (FH), are examples of conditions for which gene therapy and ...

Beam Therapeutics shows promise in gene therapy for sickle cell disease, but it also has high risks and market expectations.

We recently published a list of the 12 Biotech Stocks with the Biggest Upside Potential. In this article, we are going to take a look at where Ultragenyx Pharmaceutical, Inc. (NASDAQ:RARE) stands ...

Heart disease, particularly coronary artery disease, is a leading cause of death worldwide. In the United States alone, it ...

Fortunately, one of my learner's dads, a doctor, admitted me to the hospital. After months of scans, blood tests and minor operations, he called me into his office to share that I had another rare ...