Noonan Syndrome
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow [Phase 3]
A Phase 3 trial comparing once-weekly Somapacitan to daily Norditropin in children with growth disorders (SGA, Turner syndrome, Noonan syndr…
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CLINICAL_TRIALS research · 2026-03-19Clinical Trial Update: Constitution of a Biological Collection to Study the Pathophysiology in Noonan Syndrome
This clinical trial aims to establish a biological collection to study the pathophysiology of Noonan syndrome and is currently recruiting.
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CLINICAL_TRIALS research · 2026-03-11Clinical Trial Update: National Multicentre Study on Lipid Profile in Noonan Syndrome and Related Disorders: Trends by Age, Gender and Genotype
This clinical trial is studying lipid profiles in Noonan syndrome and related RASopathies, with analyses by age, gender and genotype; recrui…
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CLINICAL_TRIALS research · 2026-03-03Clinical Trial Update: Clinical, Genetic, and Epidemiologic Study of Children and Adults With RASopathies
Observational clinical, genetic, and epidemiologic study of children and adults with RASopathy-related conditions to characterize disease fe…
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CLINICAL_TRIALS research · 2026-02-27Clinical Trial Update: 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 [Phase 2]
Recruitment is ongoing for a Phase 2 basket study evaluating Vosoritide in children with SHOX deficiency, Turner syndrome, or Noonan syndrom…
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CLINICAL_TRIALS research · 2026-02-19Clinical Trial Update: Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial [NA]
This clinical trial update describes an internal pilot feasibility study and follow-up randomized controlled trial of Acceptance and Commitm…
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CLINICAL_TRIALS research · 2026-01-28Clinical Trial Update: Palliative Care Needs of Children With Rare Diseases and Their Families [NA]
This entry announces an ongoing study evaluating palliative care needs of children with rare diseases and their families, currently enrollin…
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CLINICAL_TRIALS research · 2026-01-21Clinical Trial Update: Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques
Clinical trial evaluating precision diagnosis and risk stratification of rare cardiomyopathies using novel cardiac MRI techniques. The study…
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CLINICAL_TRIALS research · 2025-12-18Clinical Trial Update: RASopathy Biorepository
This clinical trial update indicates that the RASopathy Biorepository is currently recruiting participants with various RASopathy-related co…
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CLINICAL_TRIALS trial_result · 2025-12-15Clinical Trial Update: Study of Metabolic Modifications in Children With Noonan Syndrome [NA]
A completed clinical trial investigating metabolic modifications in children with Noonan syndrome.
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CLINICAL_TRIALS research · 2025-12-02Clinical Trial Update: Link Between Abnormal Bleeding and Coagulation Disorders in Noonan Syndromes
A clinical trial sponsored by University Hospital, Bordeaux is investigating the link between abnormal bleeding and coagulation disorders in…
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CLINICAL_TRIALS trial_result · 2025-11-21Clinical Trial Update: A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9 [Phase 3]
Phase 3 clinical trial evaluating the safety and efficacy of somapacitan for pediatric growth disorders; results have been posted for condit…
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CLINICAL_TRIALS research · 2025-09-25Clinical Trial Update: French Registry of Children Treated With Norditropin® for Short Stature Associated With Noonan Syndrome
A French registry is enrolling children treated with Norditropin (somatropin) for short stature associated with Noonan syndrome.
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FDA approval · 2025-07-09FDA Approved: OMNITROPE — Supplemental Application (Sandoz Inc)
FDA approved a supplemental application for OMNITROPE (somatropin) submitted by Sandoz Inc.
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CLINICAL_TRIALS trial_result · 2025-04-25Clinical Trial Update: Lymphatic Phenotype in Noonan Syndrome Spectrum Disorders [NA]
A completed clinical trial studying lymphatic phenotypes in Noonan syndrome spectrum disorders.
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CLINICAL_TRIALS research · 2025-03-21Clinical Trial Update: Prevalence and Characterization of Pain in RASopathies [NA]
An ongoing clinical trial assessing how common pain is in RASopathies and describing its features across related conditions such as Costello…
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CLINICAL_TRIALS research · 2024-08-15Clinical Trial Update: MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathies [Phase 2]
A Phase 2 clinical trial is evaluating MEK inhibitors for hypertrophic cardiomyopathy in patients with RASopathies, including Noonan syndrom…
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CLINICAL_TRIALS research · 2024-08-13Clinical Trial Update: Joint and Hematologic Disorders of Noonan Syndrome: French Descriptive Cross-sectional Study
A completed French descriptive cross-sectional study characterizing joint and hematologic disorders in people with Noonan syndrome.
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CLINICAL_TRIALS trial_result · 2023-08-09Clinical Trial Update: Genetic Testing of Noonan Subjects Previously Treated With Norditropin®. An Extension to Trial GHNOO-1658 [Phase 3]
A Phase 3 clinical trial update evaluating genetic testing in Noonan syndrome patients who were previously treated with Norditropin, as an e…
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CLINICAL_TRIALS trial_result · 2023-05-11Clinical Trial Update: Treatment With HMG-COA Reductase Inhibitor of Growth and Bone Abnormalities in Children With Noonan Syndrome [Phase 3]
Phase 3 clinical trial evaluating an HMG-CoA reductase inhibitor for growth and bone abnormalities in children with Noonan syndrome has comp…
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FDA approval · 2020-01-08FDA Approved: GENOTROPIN — Supplemental Application (Pfizer Laboratories Div Pfizer Inc)
The FDA approved Genotropin (somatropin) via a supplemental application submitted by Pfizer Laboratories Div Pfizer Inc.
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CLINICAL_TRIALS trial_result · 2019-10-14Clinical Trial Update: Investigating the Long-term Efficacy and Safety of Two Doses of NN-220 (Somatropin) in Short Stature Due to Noonan Syndrome [Phase 3]
Phase 3 trial assessing long-term efficacy and safety of two doses of NN-220 (somatropin) in short stature due to Noonan syndrome has comple…
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CLINICAL_TRIALS research · 2017-10-12Clinical Trial Update: Observational Prospective Study on Patients Treated With Norditropin®
An observational prospective study assesses outcomes in patients treated with Norditropin (somatropin) for growth hormone-related disorders.
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FDA label_change · 2017-05-24FDA Approved: SEROSTIM — Supplemental Application (EMD Serono, Inc.)
FDA approved a supplemental application for SEROSTIM (somatropin) from EMD Serono, Inc. to update the product labeling. The changes expand o…
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CLINICAL_TRIALS trial_result · 2017-01-18Clinical Trial Update: Somatropin Effect on Linear Growth and Final Height in Subjects With Noonan Syndrome [Phase 3]
Phase 3 trial assessing the effect of somatropin on linear growth and final height in individuals with Noonan syndrome.