Phelan McDermid Syndrome (pms) Prevalence Tracker

POPULATION PREVALENCE ESTIMATE

13.7

per

100,000


Approximately

1 in ~7,300

individuals have PMS

10.02 - 18.60 per 100,000

95% CONFIDENCE INTERVAL

(1 in ~10,000 to 1 in ~5,400)

HOW TO ESTIMATE WHAT WAS DERIVED

Data sources

The study team reached out to clinical genetic testing laboratories, research centres, and clinical centres.

Data collected

Sited provided the number of PMS diagnosis made out of the total number of participants with autism tested at their site.

Adjustments made

Further extrapolations were made to adjust for the proportion of individuals with PMS who do not have autism, autism diagnosis age limitations, and type of genetic variant.

Population extrapolation

Centres of Disease Control estimates of autism rates were used to extrapolate to the general population.

Final estimate

After applying extrapolations, including adjustments for assay sensitivity and other factors, the final weighted average was 13.7 per 100,000.

STUDY DETAILS

Sources participating

10

Autism case evaluated

179,837

Frequency of PMS diagnosis among autism cases

1% to ~2.5%

(Raw results varied by testing method and should not be directly compared.)

Study Design

Population-based estimation study

Population represented

General population (via extrapolation from autism rates)

Key Findings

This population-based study found PMS to affect approximately 13.7 per 100,000 individuals (1 in ~7,300), with a 95% CI of 10.02-18.60 per 100,000 (1 in ~10,000 to 1 in ~5,400), substantially higher than previous estimates of 2.5-10 per million births (0.25-1 per 100,000).

Phelan-McDermid syndrome (PMS) is a genetic neurodevelopmental disorder caused by haploinsufficiency of SHANK3, either by a 22q13 deletion pr pathogenic sequence variant.