Top FDA Official Touts ‘Promise of Gene Therapy’ at MDA Annual Conference
Clinical Trial to Assess the 4-Year Efficacy of Risdiplam on SMA
Risdiplam Reduces Healthcare Resource Use and SMA-Related Complications
Onasemnogene Abeparvovec May Be an Effective Treatment Approach in SMA
Noninvasive Neuromuscular Test May Differentiate Types of SMA
More Than 1000 Plant Species Have Potential in Treating Neurodegenerative Disorders
Nusinersen May Suppress Neuroinflammation in SMA
Rare Case of an Infant With SMA and Callosal Hypoplasia Identified
Novel Drug for SMA in Phase 3 Trial Receives FDA Designation
Latest Clinical Trials
Is Scoliosis Surgery in Patients With SMA Evidence-Based?
Spinal muscular atrophy (SMA) is a disorder characterized by muscle atrophy and weakness. It is inherited in an autosomal recessive manner and is caused by mutations/deletions in the SMN1 gene, which ensures adequate levels of survival motor neuron proteins in the body. Patients with SMA tend to experience proximal muscle weakness, especially in the lower…
Presymptomatic Treatment May Benefit Patients With SMA
Studies indicate that the presymptomatic treatment of infants with spinal muscular atrophy can lead to better outcomes.
Clinical Data Demonstrate Efficacy of Nusinersen in Patients With SMA
Two clinical studies examined the efficacy of nusinersen in preventing further clinical decline in patients with SMA.
Characterizing Growth Patterns in Children With SMA
Scientists characterized the growth patterns of children with SMA type 1 or 2 using weight and supine length parameters.
Remembering Alecia Maringola, a Powerful Voice for SMA and Other Rare Diseases
Courage. Compassion. Family. Faith. Speak with anyone who knew Alecia Maringola and you’ll hear those words repeatedly, and for good reason.
Despite Living With SMA, I Have Many Thanks to Offer
Living with a rare disease, such as spinal muscular atrophy (SMA), can at times cloud the perception of being thankful.
The Ways My “Fur Baby Companion” Helps Me Cope With SMA
There are many things I use to help me cope with living with my rare disease, spinal muscular atrophy (SMA).
The Alliance for Regenerative Medicine Fights for Better Cell, Gene Therapies
The Alliance for Regenerative Medicine aims to help in the development of safe and effective regenerative therapies that can benefit rare disease patients.
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 at the first in-person annual gathering of the Muscular Dystrophy Association (MDA) since March 2019.
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.
Rare Care Podcast
An interview with SMA researcher Adrian Krainer, PhD, from the Cold Spring Harbor Laboratory.