Latest Clinical Trials
We review the role of BMI as a health measurement in general, and specifically in people with SMA type 2.
The acceleration of telemedicine during the pandemic has allowed us to gain vast insights into what works and what does not.
We share a case study of a pregnant woman with SMA type 3 and pertinent issues relating to her care.
We explore capsid-engineering gene therapy as a promising novel treatment for patients with spinal muscular atrophy, even those who are presymptomatic.
Living with spinal muscular atrophy (SMA), routine is something I greatly appreciate.
I’m immensely grateful for the opportunity to receive medication for my rare disease, spinal muscular atrophy (SMA).
Finding a sport to watch that includes people who look like me, a Duchenne muscular dystrophy (DMD) patient, is difficult.
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…
We discuss the various ethical considerations in genetic screening and gene therapy for spinal muscular atrophy (SMA).
Rare Care Podcast
An interview with SMA researcher Adrian Krainer, PhD, from the Cold Spring Harbor Laboratory.