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 various panels and heartfelt testimonies from patients and parents of children with SMA.
The event offered an opportunity for families with SMA to show the FDA the impact the disease has on patients and their caregivers, and their priorities when it comes to treatment.
The meeting was attended by 20 panelists who represented SMA patients and caregivers, ranging in age and types of SMA. Questions and discussions followed each session and more than 400 individuals took part in the meeting, either in person or through online facilities.
The key themes of the meeting included daily life with the disease, benefits and complications of surgical intervention, how the loss of swallowing affects those with type 1 SMA, the emotional and social impact on families, the need for technology to improve quality of life, and the use of necessary equipment at home.
SMA News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
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