Evidence map›Paper›PMID 37780708›Full record

ArticleFrontiers in neurology2023

Case report: A case of spinal muscular atrophy in a preterm infant: risks and benefits of treatment.

Elisa Nigro, Eyal Grunebaum, Binita Kamath, Christoph Licht, Caroline Malcolmson, Aamir Jeewa, Craig Campbell, Hugh McMillan, Pranesh Chakraborty, Mark Tarnopolsky and 1 more

Open access · goldAbstract readCase Reports
In one paragraph

Article in Frontiers in neurology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed, 1 pooled it
3.7field-weighted citation impact, top 6% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

10 citing papers in PubMed, 1 synthesis or guideline pooled it, 13 citations in OpenAlex.

  1. A systematic review of immunosuppressive protocols used in AAV gene therapy for monogenic disorders.Molecular therapy : the journal of the American Society of Gene Therapy · 2024
    Pooled it
  2. Review
  3. Article
  4. Article
  5. Article
  6. Article
  7. Article
  8. Article
  9. Clinical perspectives: Treating spinal muscular atrophy.Molecular therapy : the journal of the American Society of Gene Therapy · 2024
    Review
  10. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

11 authors at 5 institutions in 1 country.

Elisa NigroDivision of Neurology, The Hospital for Sick Children (SickKids), Toronto, ON, Canada.
Eyal GrunebaumDivision of Immunology, The Hospital for Sick Children (SickKids), Toronto, ON, Canada.
Binita KamathDivision of Gastroenterology, Hepatology and Nutrition, The Hospital for Sick Children (SickKids), Toronto, ON, Canada.
Christoph LichtDivision of Nephrology, The Hospital for Sick Children (SickKids), Toronto, ON, Canada.
Caroline MalcolmsonDivision of Hematology/Oncology, The Hospital for Sick Children (SickKids), Toronto, ON, Canada.
Aamir JeewaDivision of Cardiology, The Hospital for Sick Children (SickKids), Toronto, ON, Canada.
Craig CampbellDepartment of Pediatrics, Children's Hospital, London Health Sciences Centre, Western University, London, ON, Canada.
Hugh McMillanDepartment of Pediatrics, Children's Hospital of Eastern Ontario Research Institute, Ottawa, ON, Canada.
Pranesh ChakrabortyDepartment of Pediatrics, Newborn Screening Ontario, Children's Hospital of Eastern Ontario Research Institute, Ottawa, ON, Canada.
Mark TarnopolskyDepartment of Pediatrics, McMaster Children's Hospital, Hamilton, ON, Canada.
Hernan GonorazkyDivision of Neurology, The Hospital for Sick Children (SickKids), Toronto, ON, Canada.
SickKids Foundation · CAChildren's Hospital of Eastern Ontario · CALondon Health Sciences Centre · CAMcMaster Children's Hospital · CANewborn Screening Ontario · CA

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Spinal muscular atrophy (SMA) is a neuromuscular genetic disorder caused by the loss of lower motor neurons leading to progressive muscle weakness and atrophy. With the rise of novel therapies and early diagnosis on newborn screening (NBS), the natural history of SMA has been evolving. Earlier therapeutic interventions can modify disease outcomes and improve survival. The role of treatment in infants born preterm is an important question given the importance of early intervention. In this study, we discuss the case of an infant born at 32 weeks who was diagnosed with SMA on NBS and was treated with Spinraza

Indexed as

gene therapynewborn screeningpretermspinal muscular atrophyZolgensma

Identifiers

PMID37780708
PMCPMC10539898
OpenAlexW4386723723

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.