Crosswalk Therapeutics, Inc., a biopharmaceutical company focused on developing novel therapies for rare disorders with significant unmet medical need, today announced that it has received a research ...

In this open-label phase 1–2 study, pediatric patients with mucopolysaccharidosis type II treated with tividenofusp alfa showed substantial reductions in cerebrospinal fluid and urinary heparan ...

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 ...

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 ...

MPS II, also called Hunter syndrome, is a rare genetic disease that affects over 2,000 individuals, primarily males, world-wide, and leads to physical, cognitive, and behavioral symptoms ultimately ...

MPS II, also called Hunter syndrome, is a rare genetic disease that affects over 2,000 individuals, primarily males, world-wide, and leads to behavioral, cognitive, and physical symptoms ultimately ...

Gene therapy being developed as a one-time treatment to address the underlying cause of the disease by inserting a functional copy of the human IDUA gene into a patient’s own hematopoietic stem cells ...

Breakthrough Therapy Designation Supported by Integrated Long-Term Clinical Data Demonstrating Normalization in Cerebral Spinal Fluid Heparan Sulfate Non-Reducing End (CSF HS-NRE) U.S. FDA Confirmed ...