REGENXBIO Inc. (Nasdaq: RGNX) today announced data from its RGX-121 (clemidsogene lanparvovec) program for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, will ...

Discover Sanfilippo Syndrome a rare genetic disorder affecting childrens development Learn about its symptoms treatment ...

JCR Pharmaceuticals Co., Ltd. (TSE 4552; "JCR") announced today that it will present at the 21st Annual WORLDSymposiumtm 2025, held February 3-7, 2025, in San Diego, Calif. The poster presentations ...

Immusoft Corporation, a cell therapy company dedicated to improving the lives of patients with rare diseases, today announced that it will feature oral and poster presentations at the upcoming ...

Wedbush upped their FY2024 EPS estimates for Ultragenyx Pharmaceutical in a report released on Thursday, January 23rd. Wedbush analyst L. Chico now forecasts that the biopharmaceutical company will ...

The decision has also been welcomed by charity the MPS Society, which represents people with the ultra-rare condition, mucopolysaccharidosis type IVa (MPS IVa), or Morquio. While the decision has ...

UMB Bank n.a. increased its stake in shares of BioMarin Pharmaceutical Inc. (NASDAQ:BMRN – Free Report) by 260.1% in the 4th ...

Mucopolysaccharidosis Type II (MPS II) also known as Hunter Syndrome is when the body lacks the enzyme to break down sugar ...

Spinal cord compression (SCC) occurs in 5% to 30% of the oncology population and affects patient function, comfort, and general quality of life. Patients with lung cancer, breast cancer, prostate ...

Current price $63.06 52-week high $94.85 ...