Studying speech and swallowing development in children with SMA type 1
Part of Brain & nervous system, Genetic & congenital clinical trials.
This study looks at how speech, communication, and swallowing (“bulbar function”) develop in babies and children with SMA type 1. It aims to better understand what to expect over time for children who start approved SMA treatments early.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You (or your child) have genetic test proof of SMA (5q type).
- Symptoms started when your child was 6 months old or younger.
- Your child is between birth and 18 years old.
- Your child is already receiving an approved SMA disease-modifying treatment.
- A parent or legal guardian can understand the study, sign consent, and follow the visit plan.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This study interviews parents and families to understand their experiences with feeding and communication for children with SMA type 1. It aims to improve support and care for families.
This study uses wearable devices (like smartwatches or activity trackers) to monitor movement and motor development in young children with spinal muscular atrophy (SMA), a rare genetic condition affecting muscle strength. The goal is to see if wearables can track disease progression and treatment response better than traditional clinic visits.
This study creates a registry and clinical hub for adults with 5q spinal muscular atrophy (SMA). It aims to track health outcomes and improve care for people living with SMA.
This study is looking for children and teens with spinal muscular atrophy (SMA) who are already on a treatment like Spinraza, Evrysdi, or Zolgensma. The goal is to understand different forms of SMA and how the treatment affects them over time.
This study collects health information from people with spinal muscular atrophy (SMA) over time to learn more about the disease and how to improve care. It may help researchers understand SMA better.
This trial tests a new medicine called EXG001-307 for babies with spinal muscular atrophy (SMA) types 1 and 2. The goal is to see if it is safe and helps with muscle strength and breathing.
Hear when a new SMA1 trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.