General: 07876 682589    |   Fundraising: 07876 682589   |   Support: 0800 046 9832  
BDFA 200

BioMarin (BMRN) Says early data from Phase 1/2 BMN190 Study Shows Stabilisation of Disease. We are all very encouraged by the work of our partners in the scientific and pharmaceutical communities and hugely grateful to the courage of those parents and families who are making this happen.

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced interim results from its Phase 1/2 pivotal study for BMN 190 or cerliponase alfa, a recombinant human tripeptidyl peptidase 1 (rhTPP1), to treat of patients with late infantile CLN2 disease, a form of Batten disease. Interim data indicates that in all nine of the BMN 190 patients who have been followed for at least six months and up to 15 months, the treatment appears to show stabilisation of the disease compared to the natural history based on a standardised measure of motor and language function.

Read More Here

Make a donation:

To make a donation to the BDFA please click the button below which will take you Paypal.

 

All donations help us to provide the best support we can to families affected by Batten Disease.

 

Thank you for your support.