Latest News
Nusinersen Combined With Physical Therapy Enhances SMA Treatment
Study Finds Decreased Vertebral Height in Children With SMA
Scoliosis Surgery Leads to Favorable Outcomes in Patients With SMA
New SMA Diagnostic Method Quantifies SMN1 and SMN2 Gene Copies
Myasthenia Gravis Complicates SMA: A Rare Case
Novel Gene Therapy Leads to Better Extended Survival in SMA Model
Novel SMN Transcript Variants Shed Light on SMA Pathogenesis
FDA Approves Risdiplam for SMA in Babies Under Two Months Old
Blood MicroRNAs Useful in Monitoring Efficacy of SMA Treatment
Latest Clinical Trials
Insights
The Role of BMI as a Health Measurement in People With SMA Type 2
We review the role of BMI as a health measurement in general, and specifically in people with SMA type 2.
The Role of Telemedicine in the Treatment of Neuromuscular Disease
The acceleration of telemedicine during the pandemic has allowed us to gain vast insights into what works and what does not.
Case Study: Pregnancy in a Woman With SMA Type 3
We share a case study of a pregnant woman with SMA type 3 and pertinent issues relating to her care.
The Role of Capsid-Engineered Gene Therapy in Treatment of SMA
We explore capsid-engineering gene therapy as a promising novel treatment for patients with spinal muscular atrophy, even those who are presymptomatic.
Patient Perspectives
Routine Is an Essential Part of Managing SMA
Living with spinal muscular atrophy (SMA), routine is something I greatly appreciate.
My Life With SMA: How and Why I Dread Bloodwork
I’m immensely grateful for the opportunity to receive medication for my rare disease, spinal muscular atrophy (SMA).
How Wheelchair Power Soccer Adds Vigor, Energy to a Rare Disease Patient’s Life
Finding a sport to watch that includes people who look like me, a Duchenne muscular dystrophy (DMD) patient, is difficult.
SMA Features
The Increasingly Prominent Role of Gene Therapy in Managing SMA
We explore the genetics underpinning spinal muscular atrophy (SMA) and the merits of gene therapy in the management of patients with SMA.
The Impact of Novel SMA Therapies on Respiratory Function
How do novel therapies for spinal muscular atrophy (SMA) perform against the respiratory function decline caused by the disease?
The Challenges of Determining the Safety and Efficacy of Risdiplam in SMA
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…
Ethical Considerations in Genetic Screening and Gene Therapy for SMA
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.
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