29 Mar 2022 by AXXELIS
“A Super Fulfilling” journey – Lutz Mueller
On Tuesday 22nd March, 2022, we launched the first of this year’s series of Lightning Exchange Events, and those of us within Axxelis were delighted with what we viewed as an opening session of great clarity and impact!
Our participants were taken on what was described by our leader, Lutz Mueller, as “a super fulfilling journey.” Lutz and his team had brought the first mRNA splice modifier to market, a remarkable achievement with profound implications for treatment of the rare genetic disease: Spinal Muscular Atrophy (SMA). Throughout the session, Lutz drew on his forty years of toxicology experience, constantly sharing remarkable insights from his research into the nature and makeup of this devastating disease, and the gradual emergence of the life-changing drug, Risdiplam, now a critical weapon in the fight against SMA.
A poignant aspect of the session was an explanation by Lutz of his two-way communication sessions with the parents and family of those suffering with SMA, all of whom had been faced with the almost inevitable loss of their child before his/her second birthday. But that early loss is now giving ground to hope in the face of Risdiplam, with 93% of affected children still alive after three years of treatment, whilst maintaining good motor skills, including the ability to swallow. Infants are also achieving ‘motor milestones’ such as sitting and standing, albeit at this stage with support.
Risdiplam was developed in an astonishingly short five year period and has gone on to be available in over sixty countries around the world and has been chosen by the British Pharmacological Society for its 2022 Drug Discovery of the Year award. Your feedback is vital to our planning of future Lightning Exchange events; it enables us to improve and develop events possessing true relevance for industry professionals such as yourself. We therefore ask you to complete a brief survey regarding your experience of the recent event, for which we would be most grateful. The survey should take no longer than five minutes: https://www.axxelis.ch/event-survey/ . Many thanks!
If you were unable to attend, or would like a recap on the discussion, please access the video below for a highlighted précis of the event.
OUR NEXT EVENT
We are delighted to announce that Romain Clement , a professional software engineer & trainer from France is our presenter of the next Lightning Exchange Event, due to take place in June.