Gene therapy for rare genetic diseases continues to top the news in biotechnology. Biotechnology companies that develop and commercialize gene therapy is a rapidly growing field of medicine, especially for rare diseases. This is the tenth in a series of Blog Posts on the topic of recent business developments for gene therapy for rare diseases.
Sanfilippo (SF) Syndrome or Mucopolysaccharidosis III (MPS III) is a rare genetic metabolism disorder that prohibits the proper breakdown of the body’s sugar molecules.