The company had previously said that it expects to submit an application for approval of the therapy in the first quarter of ...

Spruce Biosciences, Inc. (Nasdaq: SPRB), a late-stage biopharmaceutical company focused on developing and commercializing novel therapies for neurological disorders with significant unmet medical need ...

Please provide your email address to receive an email when new articles are posted on . Researchers analyzed 19 children with Pompe disease seen at a Texas-based children’s hospital.

Two dose-limiting toxicities have been recorded in uniQure’s trial evaluating an investigational AAV gene therapy designed to ...

Ongoing clinical research at UNC could lead to a first-of-its-kind enzyme replacement therapy for Hunter syndrome, an ultra-rare disorder that causes progressive multisystem disease and neurologic ...

A team of researchers from Caltech and the University of Southern California has developed a microrobot for drug delivery within the body. According to its developers, the enzyme-powered microbubble ...

FDA approves avalglucosidase alfa-ngpt (Nexviazyme) for the treatment of patients 1 year and older with late-onset Pompe disease. FDA announced yesterday the approval of avalglucosidase alfa-ngpt, ...

Mitochondrial diseases are severe, often untreatable, and they leave human cells unable to grow normally without outside help. A new study published in Nature Metabolism now shows that a single gene ...

A new study led by University of Pittsburgh and UPMC Hillman Cancer Center researchers shows that an enzyme called PARP1 is involved in repair of telomeres, the lengths of DNA that protect the tips of ...

Studies into a rare nervous system disorder that causes dementia and early death in children have highlighted a possible treatment for the condition. Regular infusions of a key enzyme that is ...

The first test of in utero enzyme replacement therapy (ERT) proved safe and effective for a baby with infantile-onset Pompe disease, according to a case report. An infant with cross-reactive ...