Santhera Pharmaceuticals have shared their January update.
“Our main focus in the U.S. in the first half of the year is to bring the SIDEROS clinical trial to full enrollment and we ask the community’s help, now more than ever, to achieve this goal. The SIDEROS clinical trial, which investigates the safety and efficacy of idebenone in boys using steroids, is the only U.S. trial currently recruiting boys with DMD regardless of ambulation status and genetic mutation. The trial also has no upper age limit and is the largest clinical trial ever to study respiratory function in DMD or non-ambulatory participants. Additionally, there are provisions in place to treat non-eligible siblings when a participant is enrolled. Trial participants who have completed the trial are able to receive idebenone as part of the post-trial open-label extension period.”
January 2020 – Community Update Letter
Santhera is focussed on the development of treatments for neuromuscular and pulmonary diseases that currently lack treatment options, such as Duchenne muscular dystrophy, congenital muscular dystrophy, and cystic fibrosis.
They have dedicated more than a decade to researching potential treatments in our ongoing mission to make effective medicines available for these often overlooked, life-altering diseases. They are passionate about improving patients’ quality of life and our vision is to be a leader in providing treatments for rare diseases in these areas with high unmet medical needs.