Amicus Therapeutics announced positive interim results from the company’s CLN6 Batten disease gene therapy program.

Amicus Therapeutics inked a definitive agreement for 10 gene therapy programs licensed from Nationwide Children’s Hospital through the acquisition of Celenex.

For patients with deadly and debilitating conditions, FDA drug approvals can be cause for celebration. Many of these patients and their families have been waiting years for better therapies – or any therapy at all. They consume every public announcement about drug approvals for their particular disease like it’s water in the desert. But there is also a sense of trepidation once the drug launches. How much will this new drug cost? What will insurance cover? What will be our share?

BioMarin Pharmaceutical announced that the U.S. FDA approved Brineura (cerliponase alfa) for the rare disease, late infantile neuronal ceroid lipofuscinosis type 2 (CLN2).