Evidence map›Paper›PMID 41525567›Full record

ReviewNeurology2026

The Role of Academic Medical Centers in Personalized Experimental Therapeutic Development: Key Considerations.

Kira A Dies, Timothy W Yu, Nancy L Chamberlin, Ingrid A Holm, Alan H Beggs, Kerri O Kennedy, Judith C Fleming, Scott L Pomeroy, David K Urion, Siddharth Srivastava

Abstract readReview
In one paragraph

Review in Neurology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

10 authors.

Kira A DiesRosamund Stone Zander and Hansjoerg Wyss Translational Neuroscience Center, Boston Children's Hospital, MA.
Timothy W YuDivision of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, MA.
Nancy L ChamberlinRosamund Stone Zander and Hansjoerg Wyss Translational Neuroscience Center, Boston Children's Hospital, MA.
Ingrid A HolmDivision of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, MA.
Alan H BeggsDivision of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, MA.ORCID 0000-0001-8818-0568
Kerri O KennedyOffice of Ethics, Boston Children's Hospital, MA; and.
Judith C FlemingTranslational Research Program, Boston Children's Hospital, MA.
Scott L PomeroyDepartment of Neurology, Boston Children's Hospital, Harvard Medical School, MA.
David K UrionDepartment of Neurology, Boston Children's Hospital, Harvard Medical School, MA.
Siddharth SrivastavaRosamund Stone Zander and Hansjoerg Wyss Translational Neuroscience Center, Boston Children's Hospital, MA.ORCID 0000-0001-7008-1879

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The recent rapid rise in the number of identified genetic disorders that are potentially treatable has far outpaced the development of treatments, resulting in a worsening bottleneck in the translational pipeline. New therapeutics that target an individual's specific genetic variant, or "personalized" therapeutics, are being developed and tested at several academic medical centers. Personalized therapeutics represent a potentially transformative platform technology that could unlock new treatment models for rare disorders. At the same time, the field remains investigational, and developing and testing novel drugs in single individuals, particularly children, poses substantial sensitivities and challenges. In this review, we outline some important considerations for academic medical centers in determining their role in reviewing and supporting the development of personalized therapeutics for rare disorders, including oversight and resource allocation. We describe an institutional infrastructure and process for navigating this landscape, involving transparent oversight and communication processes.

Indexed as

Academic Medical CentersPrecision MedicineRare DiseasesHumans

Identifiers

PMID41525567
PMCPMC12805582

What Socratic holds

Textmetadata
LicenceCC BY-NC-ND
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 Socratic graph.