For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

Several companies—including JCR Pharmaceuticals, Denali Therapeutics and Regenxbio— have products in the pipeline that could ...

Hunter syndrome treatment is on a strong growth trajectory, with projections indicating an increase from $861.5 million in 2024 VANCOUVER, BRITISH COLUMBIA, CANADA ...

Dominic travels to Chicago every month to receive his treatments. Along the way, his family has connected with other families affected by Hunter syndrome, sharing their stories and offering ...

Nip­pon Shinyaku has paid $110 mil­lion up­front for the rights to sell two of Re­genxbio’s gene ther­a­pies for mu­copolysac­cha­ri­do­sis in the US and Asia. … ...

Although there's no cure for Tourette syndrome, treatments are available. Many people with Tourette syndrome don't need treatment when symptoms aren't troublesome. Tics often lessen or become ...

Enzyme replacement therapy is often used as treatment for the disorders ... to complete submission of its application for the Hunter syndrome therapy, called RGX-121, for FDA approval this ...

With an FDA approval submission for RegenXBio’s Hunter syndrome gene therapy already underway, the biopharma has now found a commercialization partner for both the U.S. and Asian markets.

One of the treatments, for a progressive disorder known as Hunter syndrome, could secure approval in the U.S. as soon as late this year. Should that happen, Regenxbio will retain rights to any ...

One of the treatments, for a progressive disorder known as Hunter syndrome, could secure approval in the U.S. as soon as late this year. Should that happen, Regenxbio will retain rights to any ...

The partnership will focus on advancing RGX-121 as a treatment for mucopolysaccharidosis II (MPS II), also known as Hunter syndrome, and RGX-111 for treating mucopolysaccharidosis I (MPS I), also ...