Roche has kicked off a worldwide early access program for its experimental therapy risdiplam by making it available to Europeans with the most serious forms of spinal muscular atrophy (SMA). Europeans with SMA type 1 will be able to get it immediately, Roche told SMA News…
An October 2019 protest by adults with spinal muscular atrophy led to an agreement under which all of Romania’s 152 known SMA patients — and those yet to be diagnosed — will gain access to Spinraza (nusinersen), Biogen’s approved treatment for this neuromuscular disease. Romanian doctors began treating…
Poland is rolling out Biogen’s spinal muscular atrophy (SMA) therapy Spinraza (nusinersen) at a faster pace than any country that has approved it, Polish experts say. Since the first injection in May 2019, about 400 of the 830 patients in Poland’s SMA registry have either started taking Spinraza…
Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest.  Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…
Russia has become the first of the 15 former Soviet republics to approve Biogen’s Spinraza (nusinersen) to treat spinal muscular atrophy (SMA). In its decision, the Russian Health Ministry noted that much more data exists on Spinraza’s benefits for patients 17 years and younger than for those 18…
Doctors have begun administering Biogen’s spinal muscular atrophy (SMA) therapy Spinraza (nusinersen) in Russia for the first time, the head of the country’s SMA patient organization reports. Forty children with the worst form of the disease — type 1 — began receiving the treatment in July under Biogen’s…
A renowned Belgian neurologist and spinal muscular atrophy (SMA) expert has become a hero in Poland for helping dozens of Polish patients obtain early access to Biogen’s Spinraza (nusinersen), the first approved treatment for the disease. Poland’s SMA community says Laurent Servais never turns a patient…
The challenges Vesna Aleksovska faced when she decided a decade ago to help fellow Macedonians with rare diseases were so daunting, they would have scared off all but the most determined. At that time, few doctors in the developing country of 2 million — now called North Macedonia — had…
A boy born in January has become North Macedonia’s first child with the most severe form of spinal muscular atrophy to make it through early infancy after he began receiving Spinraza (nusinersen), Biogen’s first approved therapy for all forms of SMA. That’s the word from Denica Velkovska, president of the…
It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing problems as Alport syndrome. Although she had known for years that something was wrong, the news that David would battle a lifelong rare disease devastated…
