On April 18, Cure SMA hosted a patient-focused drug development meeting with the Food and Drug Administration (FDA) to highlight the needs of spinal muscular atrophy (SMA) patients and their caregivers. The video shows the day in full, covering all the…
When people think of wonder drugs they think back to Alexander Fleming’s penicillin, the world’s first antibiotic. They may also think of aspirin, used as a painkiller and also as an anti-clotting agent for patients with a high risk of stroke or heart failure. But there are thousands of drugs that have…
The pharmaceutical company AveXis recently presented the latest data about its clinical trial for AVXS-101, a gene therapy for infants with type 1 SMA. The one-time infusion is designed to correct the faulty gene responsible for spinal muscular atrophy, using a harmless virus able to cross the brain-blood barrier. MORE: Gene therapy may…
Spinal muscular atrophy (SMA) is a genetic disease that causes gradual muscle wastage. Patients with SMA will have professionals from various health services as part of their healthcare team, including physical therapists. According to the American Physical Therapy Association, physical therapists will provide an essential service that focuses…
Spinal muscular atrophy (SMA) patients are susceptible to lung infections which can often lead to hospitalization. According to the American Lung Association, there are several things you can do to help prevent lung infections. Practice Good Hygiene Regularly washing hands with soap and water — particularly…
Children with spinal muscular atrophy type 1 will experience breathing difficulties and will require a personalized respiratory care plan. There are interventions that can help make breathing easier for infants with SMA type 1 and also help with feeding, fighting infections and making life more comfortable. According to…
Cure SMA has launched a series of care booklets covering a wide range of topics for SMA patients and caregivers. Their “Nutrition Basics” booklet can be downloaded as a PDF file and explains the importance of good nutrition for people with spinal…
Cure SMA has released a booklet highlighting the importance of clinical trials for research into better treatments and therapies for spinal muscular atrophy (SMA) patients. MORE: Phase 2 clinical trial of oral compound targeting genetic cause of SMA starts in Europe. The brochure explains the processes involved…
As the weather turns warmer, many families will be thinking about vacations and traveling. While traveling with a child with SMA does require some careful consideration, having SMAÂ doesn’t mean that you have to be confined to the house. The British Lung Foundation has some advice for…
Breathing issues for infants with spinal muscular atrophy (SMA) can cause serious illness and are the leading cause of death for those with type 1 and type 2 SMA. Respiratory care is essential for children with SMA. Parents will need to watch for any breathing difficulties so they can be addressed…
