The Noah’s Hope – Hope 4 Bridget family foundations have joined forces to collectively raise awareness and money for the treatment of Late Infantile Batten disease. Currently, there is no cure for the fatal, childhood disease.
The Van Houtans (Noah’s Hope) have two children who are affected; Laine age 10 and Noah~ an angel at age 11.The Kennicotts (Hope 4 Bridget) have one daughter, Bridget age 11, affected by this devastating disease.
We will always have hope for a cure. We will always have hope for our children.
The First Trial and Hopeful Treatment for Late Infantile Batten Disease!
BioMarin Pharmaceutical Inc. releases preliminary results of first ever CLN2 (Late Infantile) Batten disease trial. After the creation of a recombinant TPP1 enzyme, Ceriponase Alfa, patients in the trial had 80% reduction in clinical disease progression in one year compared to natural history (p <0.0001). The FDA and European Medicines Agency (EMA) granted Cerliponase Alfa Orphan Drug Designation. In addition, the FDA granted Cerliponase Alfa Priority Review Status and Breakthrough Therapy designation. 2017 is a very promising year for the probable treatment of CLN2. It is something we could only imagine 7 short years ago.
BioMarin’s Press Release Sept. 6, 2016
To learn more: Cerliponase_alfa_infographic_5.5