Roche Starts Global Early Access Program for SMA Therapy Risdiplam in Europe
Roche has kicked off a worldwide early access program for its experimental therapy risdiplam by making it available…
Articles by Hal Foster, PhD
Protest Secures Spinraza for All SMA Patients in Romania
An October 2019 protest by adults with spinal muscular atrophy led to an agreement under which all of Romania’s…
Nearly Half of Poland’s SMA Patients on Track to Get Spinraza, Experts Say
Poland is rolling out Biogen’s spinal muscular atrophy (SMA) therapy Spinraza (nusinersen) at a faster pace than any…
‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases
Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their…
Russia Approves Spinraza to Treat All SMA Patients
Russia has become the first of the 15 former Soviet republics to approve Biogen’s Spinraza (nusinersen) to treat …
40 Russian Children Now on Spinraza, as Moscow-based Advocacy Group Pushes for More Treatment
Doctors have begun administering Biogen’s spinal muscular atrophy (SMA) therapy Spinraza (nusinersen) in Russia for the first time, the…
Belgian Neurologist Led Efforts to Bring Spinraza to Polish Kids with SMA
A renowned Belgian neurologist and spinal muscular atrophy (SMA) expert has become a hero in Poland for helping dozens…
Macedonian Gaucher Activist Publicizes Plight of Rare Disease Patients
The challenges Vesna Aleksovska faced when she decided a decade ago to help fellow Macedonians with rare diseases were so…
Spinraza Gives Baby with Type 1 SMA in North Macedonia Hope for Healthy Life
A boy born in January has become North Macedonia’s first child with the most severe form of spinal muscular atrophy…
World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients
It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his…