Latest Clinical Trials
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 early next year at the first in-person annual gathering of the Muscular Dystrophy Association (MDA) since March 2019. The 2022 MDA Clinical & Scientific Conference, set for March 13-16 in Nashville, Tennessee, is expected to attract between 700 and 800 people in…
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.
Several recently published studies may help enhance our treatment of spinal muscular atrophy (SMA). Review key points of some of them below. Evaluating a Novel Myostatin Inhibitor as an SMA Treatment A new article in Advances in Therapy describes a phase 1 trial of a monoclonal antibody intervention being developed as a treatment for SMA1.…
Living with spinal muscular atrophy (SMA) can not only weaken your physical and mental health, but also weighs heavily on romantic relationships.
Finding in-home, quality care is an ongoing and very tiring process for one living with spinal muscular atrophy (SMA).
Living with spinal muscular atrophy (SMA) can trigger anxiety, and it’s an inescapable enemy most of the time.
SMA Clinical Insights
We explore capsid-engineering gene therapy as a promising novel treatment for patients with spinal muscular atrophy, even those who are presymptomatic.
We explore the genetics underpinning spinal muscular atrophy (SMA) and the merits of gene therapy in the management of patients with SMA.
How do novel therapies for spinal muscular atrophy (SMA) perform against the respiratory function decline caused by the disease?
In recent years, one of the great leaps in medical advancements has been the development of drugs such as risdiplam, which is used to treat spinal muscular atrophy (SMA). It is the first drug to be approved as a survival motor neuron 2 (SMN2) mRNA splicing modifier. Dhillon did an excellent job describing the key…
Rare Care Podcast
An interview with SMA researcher Adrian Krainer, PhD, from the Cold Spring Harbor Laboratory.