Bardet-biedl Syndrome
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Sign up freeTop drugs studied for this disease
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FDA approval · 2026-03-19FDA Approved: IMCIVREE — Supplemental Application (Rhythm Pharmaceuticals, Inc)
FDA approved a supplemental application for IMCIVREE (setmelanotide) to expand its indications for genetic obesity disorders. The approval b…
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CLINICAL_TRIALS research · 2025-09-29Clinical Trial Update: Early Genetic Identification of Obesity
This clinical trial update describes ongoing research on early genetic identification of obesity, highlighting genetic conditions such as Ba…
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CLINICAL_TRIALS trial_result · 2025-08-07Clinical Trial Update: Characterizing the Genotype and Phenotype in Adults With Bardet-Biedl Syndrome
This completed clinical trial reports on characterizing genotype and phenotype in adults with Bardet-Biedl syndrome. It provides observation…
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CLINICAL_TRIALS research · 2025-06-13Clinical Trial Update: ARPKD Database Study
ClinicalTrials.gov entry indicates that the ARPKD Database Study sponsored by Children’s Hospital of Philadelphia is currently recruiting, a…
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CLINICAL_TRIALS research · 2025-03-27Clinical Trial Update: Inherited Retinal Degenerative Disease Registry
A Foundation Fighting Blindness–sponsored inherited retinal degenerative disease registry is currently recruiting participants to collect in…
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CLINICAL_TRIALS trial_result · 2024-11-26Clinical Trial Update: Phase 3 Crossover Trial of Two Formulations of Setmelanotide in Participants With Specific Gene Defects in the MC4R Pathway [Phase 3]
Rhythm Pharmaceuticals reports results from a Phase 3 crossover trial comparing two formulations of setmelanotide in participants with Barde…
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CLINICAL_TRIALS research · 2024-09-27Clinical Trial Update: Bardet Beidle Syndrome in a Syrian Adolescent : a Rare Case Report
A rare case report of Bardet-Biedl syndrome in a Syrian adolescent, with the trial registry indicating the study is not yet recruiting.
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CLINICAL_TRIALS trial_result · 2024-07-10Clinical Trial Update: Setmelanotide in Pediatric Participants With Rare Genetic Diseases of Obesity [Phase 3]
Phase 3 trial update reports results for setmelanotide in pediatric patients with rare genetic obesity conditions, including Bardet-Biedl Sy…
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CLINICAL_TRIALS trial_result · 2023-12-01Clinical Trial Update: Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity [Phase 3]
Phase 3 trial of setmelanotide (MC4R agonist) in Bardet-Biedl and Alström syndromes with obesity has completed with results posted in 2023.
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CLINICAL_TRIALS trial_result · 2008-08-08Clinical Trial Update: Bardet-Biedl Syndrome Study: Clinical and Genetic Epidemiology Study in Adults
This is a completed clinical and genetic epidemiology study in adults with Bardet-Biedl syndrome conducted at University Hospital, Strasbour…