Cure SMA Hosts SMA Patient-Focused Drug Development Meeting With FDA

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by Wendy Henderson |

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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.

MORE: The importance of clinical trials for SMA patients

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.

MORE: Gene therapy may help babies with type 1 SMA

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