Keyword: BioMarin Pharmaceutical

While BioMarin has filed its hem A gene therapy valrox for approvals in the U.S. and EU, Roche is only running a phase 3 lead-in study for SPK-8011.

Gene and cell therapies will make strides in 2020, but their developers will have to address challenges in manufacturing, reimbursement and more.

Previously, despite repeated delays, tendered Spark shares stayed relatively stable at around 20%—until now.

BioMarin is collaborating again with Believe Limited for an arts program to support people with hemophilia.

BioMarin and Invitae are expanding their free genetic testing program for children with seizures with new partners and a wider patient age range.

BioMarin and NICE have been negotiating for a year, but that hasn’t helped the company secure reimbursement for its Batten disease drug.

M&A will help the biopharma industry deal with significant new challenges, Morningstar said, including pressure from lawmakers and payers to lower prices.

The BioMarin-sponsored musical created by teens with hemophilia debuts today after a weekend of practice, camaraderie and education.

BioMarin is already expanding its manufacturing site in Ireland, adding more than 50 jobs in the process.

BioMarin and Believe Limited are helping 25 high schoolers create the first-ever musical about bleeding disorders.