The Alliance for Regenerative Medicine aims to help in the development of safe and effective regenerative therapies that can benefit rare disease patients.
Five years after the approval of nusinersen (Spinraza®) for SMA, Adrian Krainer, PhD, is immensely proud of his role in developing the lifesaving drug.
Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA) will take center stage at the first in-person annual gathering of the Muscular Dystrophy Association (MDA) since March 2019.
Sofijka and Lukyan Radysh are among an estimated 300 to 400 SMA patients in Ukraine, Europe’s poorest country as measured by annual per-capita income.
Read about the latest clinical insights into improving best practices for treating spinal muscular atrophy.
There have been several recent publications related to spinal muscular atrophy (SMA). Below is a summary of some pieces that add to our understanding of the disease. Identifying Modifiers of SMN Proteins A study out of Bethesda, Maryland, published in Cell Reports, aimed at investigating the mechanisms by which survival motor neuron (SMN) proteins are…
When it comes to rare disorders, Israel—a New Jersey-sized nation of 9.3 million that’s made headlines this year for its highly successful coronavirus vaccination campaign—is clearly an emerging powerhouse.
When Gennadiy Ilyashenko’s daughter, Sophia, was born, she looked like a healthy, happy baby. But after Sophia failed to meet certain milestones, like being able to sit up by herself, Ilyashenko took her to the family’s London, England pediatrician, who told him “she’s just a lazy baby, there’s nothing wrong with her.” Yet Sophia’s condition…