Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate | The Independent
The Independent has covered the story of 21-year-old Camden Alderman, who is unable to receive a new gene therapy that could cure his rare condition because it is not profitable enough for the pharmaceutical industry to develop & take it to market.
This is an important and growing problem affecting children with rare conditions across Europe. CHA colleagues are working hard to advocate for a level playing field for paediatric drugs and devices, to overcome the regulatory, licensing and commercial barriers that slow down innovation for rare conditions and to seek government support to drive pharma and industry investment in paediatric research.