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Bardet-Biedl Syndrome

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December 24, 2024
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FDA expands setmelanotide approval for rare forms of obesity to children aged 2 to 5 years

FDA expands setmelanotide approval for rare forms of obesity to children aged 2 to 5 years

The FDA approved an expanded indication for setmelanotide to include children aged 2 to 5 years with several genetic forms of obesity, Rhythm Pharmaceuticals announced.

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October 26, 2023
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People with Bardet-Biedl syndrome lower metabolic syndrome score with setmelanotide

People with Bardet-Biedl syndrome lower metabolic syndrome score with setmelanotide

DALLAS — Setmelanotide may lower the risk for metabolic syndrome for people with a rare monogenic form of obesity, according to data presented at ObesityWeek.

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May 01, 2023
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Setmelanotide reduces hyperphagia, improves quality of life in Bardet-Biedl syndrome

Setmelanotide reduces hyperphagia, improves quality of life in Bardet-Biedl syndrome

Treatment with the melanocortin-4 receptor agonist setmelanotide reduced hyperphagia and led to improvements in emotional well-being among children and adults with Bardet-Biedl syndrome, according to study findings.

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November 28, 2022
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Setmelanotide significantly reduces body weight for people with Bardet-Biedl syndrome

Setmelanotide significantly reduces body weight for people with Bardet-Biedl syndrome

Setmelanotide led to significant reductions in weight among people with obesity and the genetic disorder Bardet-Biedl syndrome, but not Alström syndrome, according to findings published in The Lancet Diabetes & Endocrinology.

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June 17, 2022
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FDA approves setmelanotide for patients with Bardet-Biedl syndrome

FDA approves setmelanotide for patients with Bardet-Biedl syndrome

The FDA has cleared a melanocortin-4 receptor agonist for chronic weight management in people with a rare genetic disease of obesity, according to an industry press release.

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July 13, 2020
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Rare obesity disorders call for genetic testing, targeted therapies

Rare obesity disorders call for genetic testing, targeted therapies

Severe obesity starting early in life is a hallmark of rare genetic disorders of obesity. A genetic diagnosis can alleviate feelings of guilt or blame and lessen social stigma for youths with these disorders and their families.

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September 18, 2019
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Ocugen gene therapy receives orphan drug designation

OCU400, a gene therapy candidate, has received a second orphan drug designation from the FDA for the treatment of CEP290 mutation associated retinal disease, Ocugen announced in a press release.