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About 175 results

ALLMedicine™ Alström Syndrome Center

Research & Reviews  66 results

Spinal fusion with motor evoked potential monitoring using remimazolam in Alström syndr...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8615471
Medicine Arashiro A, Shinzato H et. al.

Dec 30th, 2021 - Alström syndrome is a rare genetic disorder characterized by obesity, diabetes mellitus, cardiomyopathy, and liver dysfunction. Further, scoliosis, a common symptom of Alström syndrome, often requires surgical intervention for functional impairmen...

The Hedgehog Signaling Pathway is Expressed in the Adult Mouse Hypothalamus and Modulat...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8482854
ENeuro Antonellis PJ, Engle SE et. al.

Sep 19th, 2021 - The hedgehog signaling pathway is best known for its role in developmental patterning of the neural tube and limb bud. More recently, hedgehog signaling has been recognized for its roles in growth of adult tissues and maintenance of progenitor cel...

Translational readthrough of ciliopathy genes BBS2 and ALMS1 restores protein, ciliogen...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8353411
EBioMedicine Eintracht J, Forsythe E et. al.

Aug 9th, 2021 - Ciliary dysfunction underlies a range of genetic disorders collectively termed ciliopathies, for which there are no treatments available. Bardet-Biedl syndrome (BBS) is characterised by multisystemic involvement, including rod-cone dystrophy and r...

Anesthetic Considerations in Alström Syndrome: A Case Report.
https://doi.org/10.1213/XAA.0000000000001503
A&A Practice; Aslam MZ, O'Meachair A et. al.

Jul 21st, 2021 - Alström syndrome is a rare genetic condition that affects cardiac, respiratory, endocrine, hepatorenal, gastrointestinal, auditory, ophthalmic, and musculoskeletal systems. This multisystem syndrome poses significant anesthetic challenges, along w...

Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Patients With Moderate to Severe Obesity
https://clinicaltrials.gov/ct2/show/NCT03746522

Jul 20th, 2021 - This pivotal, phase 3 study is designed to confirm the efficacy and safety of setmelanotide, a potent melanocortin receptor type 4 (MC4R) agonist, for the treatment of obesity and hyperphagia in patients with Bardet Biedl syndrome (BBS) or Alström...

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Clinicaltrials.gov  2 results

Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Patients With Moderate to Severe Obesity
https://clinicaltrials.gov/ct2/show/NCT03746522

Jul 20th, 2021 - This pivotal, phase 3 study is designed to confirm the efficacy and safety of setmelanotide, a potent melanocortin receptor type 4 (MC4R) agonist, for the treatment of obesity and hyperphagia in patients with Bardet Biedl syndrome (BBS) or Alström...

Clinical Study of a Single Ciliopathy: Alström Syndrome
https://clinicaltrials.gov/ct2/show/NCT02890550

Jun 21st, 2019 - The aim of the study is to characterize the clinical manifestations of ALMS within the ciliopathies to prevent complications and determine preventive and therapeutic targets. The investigators believe that the clinical consequences of mutations in...

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News  2 results

Imcivree Considered for Expanded Label; Semglee Hits Shelves; Buphenyl for T2D?
https://www.medpagetoday.com/endocrinology/generalendocrinology/95785

Nov 21st, 2021 - The FDA accepted a priority review of a supplemental new drug application for setmelanotide (Imcivree), a melanocortin-4 receptor agonist, for the treatment of Bardet-Biedl syndrome and Alström syndrome, Rhythm Pharmaceuticals announced. The FDA a...

BRIEF-Rhythm Pharmaceuticals Reports Q4 Net Loss Per Common Share $0.78
https://www.reuters.com/article/brief-rhythm-pharmaceuticals-reports-q4/brief-rhythm-pharmaceuticals-reports-q4-net-loss-per-common-share-0-78-idUSASA00FZ2

Mar 2nd, 2020 - March 2 (Reuters) - Rhythm Pharmaceuticals Inc: * RHYTHM PHARMACEUTICALS REPORTS FOURTH QUARTER AND FULL YEAR 2019 FINANCIAL RESULTS * RHYTHM PHARMACEUTICALS - EXPECTS EXISTING CASH, EQUIVALENTS & SHORT-TERM INVESTMENTS WILL ENABLE CO TO FUND OPER...

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