Study examines how to better support families of infants with fatal muscle disease

NCT ID NCT01862042

First seen Jun 26, 2026 · Last updated Jun 26, 2026 · Updated 1 time

Summary

This study looked at the quality of supportive and palliative care for infants under one year old with spinal muscular atrophy (SMA) type 1, a severe genetic muscle disease. Researchers followed 39 infants and asked families to keep a diary about care. One year after the child's death, parents completed a questionnaire with a psychologist to share their experiences and challenges. The goal was to understand what works well and what could be improved in end-of-life care for these infants.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

What this could lead to
If successful, this study could help improve how doctors and families manage care for infants with SMA type 1, making the experience more comfortable and supportive.
What could go wrong
This is a small, observational study with only 39 participants and no new treatment being tested. Results may not apply to all patients or change standard care.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Necker Hospital

    Paris, 75015, France

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Other studies related to the condition(s) this trial covers.