A Phase 2 Basket Study of Vosoritide in Children With Turner Syndrome, SHOX Deficiency and Noonan Syndrome With an Inadequate Response to Human Growth Hormone

Inclusion Criteria:

1. Participants must be ≥ 3 years old, and < 10 years old (females) or < 11 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 ≤ -2.00 SDs in reference tothe general population of the same age and sex.

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

5. Have been receiving continuous hGH for the treatment of short stature associatedwith their condition for a minimum of 1 year immediately prior to enrollment.

6. Are willing to continue on hGH for the Baseline Growth Phase, and for 2 years postrandomization if randomized to the hGH arm.

7. Inadequate response to prior hGH treatment.

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. Congenital heart disease which places the participant at increased risk of anadverse 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.