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.
SMA Insights
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.
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).
The Curious Link Between Spinal Muscular Atrophy and Fatty Acid Metabolism
A group of researchers recently examined the fascinating link between spinal muscular atrophy (SMA) and defective fatty acid metabolism.
Therapeutic Frontiers in Managing Spinal Muscular Atrophy Type 1
The thrust of medical progress in treating SMA type 1 is now to bring it into the realm of rare but manageable diseases.
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