Category: rare diseases

Trump Tariffs Threaten Rare Disease Cell and Gene Therapy

The biotech industry is sounding the alarm as Trump’s tariffs put rare disease cell and gene therapy at risk. With over 10,000 rare diseases still lacking FDA-approved therapies, companies like BioMarin are urging the preservation of incentives to sustain innovation. BioMarin, known for gene therapies like Roctavian for hemophilia A, is facing financial challenges despite […]