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

Mepsevii is approved to treat Mucopolysaccharidosis VII ... in upcoming commercial launches and advancing multiple phase III programs, which are expected to reduce net cash used in operations ...

Ultragenyx Pharmaceutical RARE reported first-quarter 2025 loss of $1.57 per share, wider than the Zacks Consensus Estimate of a loss of $1.54. The company had incurred a loss of $2.03 per share in ...

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.

Mucopolysaccharidosis VII (Sly syndrome ... 76 More recently a phase I/II clinical study was undertaken in UK involving 3 patients and a phase III study involving 12 patients in the USA is now ...

It also develops UX701 that is in Phase 2 clinical trial for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has ...

Simpson, NASDAQ: RGNX) for RGX-121 (clemidsogene lanparvovec), a potential first-in-class, investigational gene therapy for the treatment of Mucopolysaccharidosis II (MPS II). The FDA granted ...

In May 2025, Denali Therapeutics Inc. announced a Phase 2/3 study of the efficacy and safety of tividenofusp alfa (DNL310), ...

And his character is kind of a big deal. By Tony Maglio [This story contains spoilers for the season four finale of Power Book III: Raising Kanan.] The good: Shameik Moore has been cast as ...

Luther Burden III, Ozzy Trapilo, and Shemar Turner address the following day one of Rookie Minicamp. Second round picks Luther Burden III, Ozzy Trapilo and Shemar Turner meet the media Saturday at ...