The Roctavian program was originally acquired from University College London and appears to have limited commercial potential due to ongoing reimbursement issues in the US and EU. BioMarin has ...

With grant funding from governments at multiple levels in Canada, Entos Pharmaceuticals is looking to plant roots at a CAD $198.5 million ($137.2 million) R&D and manufacturing hub in Edmonton. | The ...

Jessica Rich, nine, has a very rare and serious illness called Batten disease, but her life-saving drug could be about to be ...

BioMarin’s treatment looks to be truly groundbreaking ... professor of haemostasis and thrombosis at Barts and the London School of Medicine and Dentistry and haemophilia clinical director ...

As Rare Disease Day is commemorated on 28 February, experts discuss the barriers to gene therapy access for some rare diseases.

Currently, patients are commonly treated with Biomarin Pharmaceuticals’ Kuvan (sapropterin dihydrochloride), an enzyme cofactor therapy, or Palynziq (pegvaliase), an enzyme replacement therapy.

Alexander Hardy; President, Chief Executive Officer, Director; Biomarin Pharmaceutical Inc Brian Mueller; Chief Financial Officer, Executive Vice President; Biomarin Pharmaceutical Inc Cristin ...