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Does your child have Noonan syndrome and isn’t growing well?
Noonan syndrome is a genetic condition that affects boys and girls equally, and occurs in 1:1000 to 1:2500 live born children. Children with Noonan syndrome may have facial features that include low-set or abnormally shaped ears, sagging eyelids, wide set and/or down-slanted eyes, extra fold of skin above the eyes, and a small jaw. These typical facial features change with age, sometimes making Noonan syndrome difficult to diagnose. Webbing of the neck tissue and breastbone abnormalities are also common. Heart defects or disease is present at birth in 50% of patients with Noonan syndrome. Approximately 80% of children with Noonan syndrome are shorter than average children of the same age and sex. About half of adults with Noonan syndrome have short stature compared to other adults.
There is no single treatment for the short stature affecting children with Noonan syndrome. Although not FDA approved for this use, recombinant human growth hormone (rhGH) is sometimes used to treat short children with Noonan syndrome. However, recently published reports suggest catch-up growth with rhGH may not be as good as expected.
Insmed Inc. is starting a research study in Noonan syndrome with a different type of growth-promoting treatment. The rationale for researching this particular therapy is that it bypasses a problem that may affect response to growth hormone in children with Noonan syndrome. The study is being carried out to see if giving the study drug for one year will improve growth in eligible children with growth failure due to Noonan syndrome.
Your child may qualify for the study if they:
- Have been diagnosed with Noonan syndrome or are suspected of having Noonan syndrome
- Are below a certain height
- Are at least 2 years old and not yet in puberty
- Are not currently being treated with recombinant human growth hormone
If you are interested in finding out more about our clinical study please talk with your physician and share this information with them, or you can contact Insmed Incorporated at clinicaltrials@insmed.com or 1-866-464-7539 to obtain information on the nearest participating physician.
Insmed Incorporated is a biopharmaceutical company focused on the discovery and development of drug candidates for the treatment of metabolic diseases and endocrine disorders where there are unmet medical needs. For more information, please visit www.insmed.com.
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