News

Thank you Joanne! I pray that God meets all of Zack and your needs as well. Blessings!…

Ari, you have been through it! We will continue to pray that all goes well with your respiratory status and the next procedure to get rid of the rest of the stones. Blessings to you!…

Higher levels of the glial fibrillary acidic protein (GFAP) in the fluid around the brain and spinal cord are associated with poorer motor function among people with spinal muscular atrophy (SMA), a new study reports. These results indicate that cells called astrocytes are more active in advanced SMA,…

In the first year of a newborn screening program for spinal muscular atrophy (SMA) in Japan, more than 10,000 babies were screened and one tested positive for the disease, according to a new study. The baby with SMA was able to receive the gene therapy Zolgensma (onasemnogene…

Compared to what would be expected without treatment, the approved oral therapy Evrysdi (risdiplam) led to marked benefits in survival and motor outcomes for babies with spinal muscular atrophy (SMA) type 1, according to a comparison of clinical trial data. “Untreated infants with Type 1 SMA ……

Compared to what would be expected without treatment, the approved oral therapy Evrysdi (risdiplam) led to marked benefits in survival and motor outcomes for babies with spinal muscular atrophy (SMA) type 1, according to a comparison of clinical trial data. “Untreated infants with Type 1 SMA ……

Moxifloxacin, an antibiotic with a well-established safety profile, boosted production of the SMN protein in cellular models of spinal muscular atrophy (SMA) and it extended the lifespan and improved motor function in mice with the disease, a new study reports. “Preclinical data from patient-derived MNs [motor neurons] and…

Initiating a disease-modifying therapy (DMT) before the onset of symptoms and at the earliest time possible results in the best outcomes in infants with spinal muscular atrophy (SMA), with most achieving otherwise unachievable motor milestones and within the normal age range. This is particularly crucial for infants…

For most of his life, Jose Flores avoided interacting with the spinal muscular atrophy (SMA) community because he felt out of touch with it. An SMA type 4 patient, Flores didn’t need a wheelchair until he was 22, and found it difficult emotionally to see children who…

Since beginning Michael’s physical therapy in October 2017, we have been focusing on improving flexibility and increasing stability in his core musculature. This has greatly improved Michael’s ability to ascend and descend ramps with improved stability and has reduced his pain while sitting throughout the day in his wheelchair. Also,…