New publication: Two decades of discovery: SMA research, from darkness to hope by Rare Revolution Magazine.
We are delighted to invite you to read the latest Two decades of discovery: SMA research, from darkness to hope by Rare Revolution Magazine.
Rare Revolution Magazine has featured Two decades of discovery: SMA research, from darkness to hope written by Becky Pender, RARE Revolution Magazine, interviewed with Professor Dr. Brunhilde Wirth, clinical geneticist and retired chair of the scientific advisory board (SAB) for SMA Europe, Thomas Koed Doktor, chair of the treatment committee and a person living with spinal muscular atrophy (SMA), and Stefania Corti, professor of neurology at the University of Milan, and the incoming SAB chair for SMA Europe.
In just 20 years, SMA has gone from a condition with very limited hope to one with multiple approved therapies.
Breakthroughs in understanding SMN1, SMN2, and splicing opened the door to treatments that have transformed lives.
But today, we are in a new phase of ensuring access for everyone, improving long-term outcomes and continuing research beyond what exists.
Progress is real, but the work is not finished.
Huge thanks to Rare Revolution Magazine and all who contributed. Together, #WeAreOne.