[Wiedemann-Rautenstrauch syndrome. The first description of a clinical case in the Russian Federation].


Journal

Problemy endokrinologii
ISSN: 2308-1430
Titre abrégé: Probl Endokrinol (Mosk)
Pays: Russia (Federation)
ID NLM: 0140673

Informations de publication

Date de publication:
08 Oct 2023
Historique:
received: 13 09 2023
accepted: 08 10 2023
revised: 04 10 2023
medline: 26 5 2024
pubmed: 26 5 2024
entrez: 26 5 2024
Statut: epublish

Résumé

Wiedemann-Rautenstrauch syndrome (neonatal progeroid syndrome) is an ultra-orphan disease from the group of premature aging syndromes with an autosomal recessive type of inheritance associated with mutations in the POLR3A, POLR3B, and POLR3GL genes encoding RNA polymerase III. The incidence of the disease is currently unknown. We present the first clinical description in Russian Federation of a patient 7 years 6 months old with Wiedemann-Rautenstrauch syndrome (compound heterozygous mutations in POLR3A gene) with progeroid features, adentia, growth retardation (height SDS -3,41, height velocity SDS -2,47), underweight (BMI SDS -6,20), and generalized lipodystrophy. The article presents the observation of the patient for 1.5 years, the world experience of dynamic follow-up of patients with neonatal progeroid syndrome, differential diagnosis, as well as recommendations for the management of patients with this syndrome. Given the lack of specific treatment to date, patients are observed by a multidisciplinary team of physicians.

Identifiants

pubmed: 38796765
doi: 10.14341/probl13369
doi:

Substances chimiques

RNA Polymerase III EC 2.7.7.6
POLR3A protein, human EC 2.7.7.6

Types de publication

Journal Article Case Reports

Langues

rus

Sous-ensembles de citation

IM

Pagination

86-93

Auteurs

A L Kungurtseva (AL)

I.M. Sechenov First Moscow State Medical University (Sechenov University).

A V Popovich (AV)

I.M. Sechenov First Moscow State Medical University (Sechenov University).

Y V Tikhonovich (YV)

I.M. Sechenov First Moscow State Medical University (Sechenov University).

A V Vitebskaya (AV)

I.M. Sechenov First Moscow State Medical University (Sechenov University).

Articles similaires

[Redispensing of expensive oral anticancer medicines: a practical application].

Lisanne N van Merendonk, Kübra Akgöl, Bastiaan Nuijen
1.00
Humans Antineoplastic Agents Administration, Oral Drug Costs Counterfeit Drugs

Smoking Cessation and Incident Cardiovascular Disease.

Jun Hwan Cho, Seung Yong Shin, Hoseob Kim et al.
1.00
Humans Male Smoking Cessation Cardiovascular Diseases Female
Humans United States Aged Cross-Sectional Studies Medicare Part C
1.00
Humans Yoga Low Back Pain Female Male

Classifications MeSH