A Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

Inclusion Criteria:

1. Participants must be ≥ 3 years old, and < 11 years old (females) or < 12 years old (males), at the time of signing the informed consent form

2. A genetically confirmed diagnosis of Turner syndrome, SHOX deficiency or Noonansyndrome.

3. A height assessment corresponding to a height Z-score of ≤ -1.28 SDs (below the 10thpercentile for height) in reference to the general population of the same age andsex.

4. Tanner Stage 1, at time of signing the ICF.

5. Previous or current hGH treatment for short stature associated with their condition.

6. Inadequate growth confirmed with an AGV that is less than age- and sex-matchedaverage stature AGV determined using median heights from CDC growth charts

Exclusion Criteria:

1. Participants with Turner syndrome known to have Y-chromosome material unless theyhave undergone gonadectomy and have fully external female genitalia.

2. Diagnosis of systemic disease or condition that may cause short stature other thanTurner syndrome, SHOX deficiency, or Noonan syndrome, eg, renal, neoplastic,pulmonary, cardiac, gastrointestinal, immunologic and metabolic disease.

3. Bone age advanced beyond chronological age by more than 2 years.

4. Uncorrected congenital heart disease which places the participant at increased riskof an adverse cardiac outcome in the setting of hypotension,

5. Have an unstable condition likely to require surgical intervention during the study.

6. Evidence of decreased growth velocity (AGV < 1.5 cm/year) as assessed over a periodof at least 6 months and growth plate closure assessed using bilateral lowerextremity X-rays.

7. Previous limb-lengthening surgery, or planned or expected to have limb lengtheningsurgery during the study period.

8. Planned or expected bone-related surgery (ie, surgery involving disruption of bonecortex, excluding tooth extraction), during the study period.