Latest News
New Insights on Preferred SMA Treatments From Patients and Caregivers
Abnormal Methylation Patterns May Disrupt Gene Function in SMA
Clear Panel Design Criteria Can Ensure Accuracy of LCFAOD Diagnosis
In Pretreatment Era, SMA Increased Mortality Rates Across Age Groups
Risdiplam Significantly Improves SMA Motor Function in Phase 3 Trial
Lifetime Mobility Patterns Associated With Quality of Life in 5q-SMA
Review Finds Lack of Health State Utility Values for Patients With SMA
French Registry of Spinal Muscular Atrophy Shows Encouraging Results
Early Treatment of SMA May Be More Important Than Combination Therapy
Latest Clinical Trials
Features
The Brains Behind Nusinersen: A Visit With Geneticist Adrian Krainer, PhD
Five years after the approval of nusinersen (Spinraza®) for SMA, Adrian Krainer, PhD, is immensely proud of his role in developing the lifesaving drug.
MDA 2022 Conference to Focus on Promising Therapies for SMA, DMD
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…
SMA Patients, Families Struggle in War-Torn Ukraine, Europe’s Poorest Country
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.
Treating Spinal Muscular Atrophy: Recent Clinical Insights
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.…
Patient Perspectives
Living With SMA Can Challenge the Health, Stability of Married Couples
Living with spinal muscular atrophy (SMA) can not only weaken your physical and mental health, but also weighs heavily on romantic relationships.
In-Home Care For SMA Patients Lacks Support
Finding in-home, quality care is an ongoing and very tiring process for one living with spinal muscular atrophy (SMA).
Growing Up With SMA: Fear, Confusion, and Anxiety
Living with spinal muscular atrophy (SMA) can trigger anxiety, and it’s an inescapable enemy most of the time.
SMA Clinical Insights
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…
Rare Care Podcast
An interview with SMA researcher Adrian Krainer, PhD, from the Cold Spring Harbor Laboratory.
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