Promising Mucopolysaccharidosis Type I Pipeline Therapies such as Laronidase, Antihistamine, Antipyretic, JR-171, AGT-181, ...

JCR Pharmaceuticals Co., Ltd. (TSE 4552; "JCR") announced today that it will present updated preclinical data from its proprietary J-Brain Cargo®-applied adeno-associated virus (AAV) vector gene ...

Data from the first patients enrolled into Regenxbio's trial of its gene therapy for rare inherited disease mucopolysaccharidosis type I (MPS I) – also known as Hurler syndrome – has shown the ...

Hurler- Scheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type A and B), and more. JCR strives to expand the possibilities for patients while accelerating medical ...

The FDA will issue a decision on the BLA for RGX-121 by November, which if positive, could make it the first gene therapy for Hunter syndrome.

SEWARD, Neb. (KOLN) - A Seward fifth grader battling a rare disease known as MPS, or Hurler Syndrome, was honored by her classmates Thursday. Following a proclamation from Governor Jim Pillen ...

Prader-Willi syndrome is a rare genetic disease that causes poor feeding in infancy but later triggers insatiable hunger.

A newly developed AI can predict which cancer patients are at risk for a life-threatening wasting syndrome, a new study says. The syndrome, called cachexia, accounts for about 20% of all cancer ...

The Milwaukee Brewers opted to not bring back a starting pitcher from the last few years this past offseason. Colin Rea was a solid piece for Milwaukee over the last few years. He signed a minor ...