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About 205 results

ALLMedicine™ Alström Syndrome Center

Research & Reviews  75 results

PATAS, a First-in-Class Therapeutic Peptide Biologic, Improves Whole-Body Insulin Resis...
https://doi.org/10.2337/db22-0058
Diabetes Schreyer E, Obringer C et. al.

Jul 14th, 2022 - Adipose tissue is a key regulator of whole-body metabolic fitness because of its role in controlling insulin sensitivity. Obesity is associated with hypertrophic adipocytes with impaired glucose absorption, a phenomenon existing in the ultrarare m...

Identification of ALMS1 pathogenic variants in Chinese patients with Alström syndrome.
https://doi.org/10.1080/13816810.2022.2092759
Ophthalmic Genetics; Huang L, Guo M et. al.

Jul 6th, 2022 - Identification of ALMS1 pathogenic variants in Chinese patients with Alström syndrome.|2022|Huang L,Guo M,Zhou Y,Liang T,Li N,|

Hearing Loss in Adults With Alström Syndrome-Experience From the UK National Alström Se...
https://doi.org/10.1097/MAO.0000000000003553
Otology & Neurotology : Official Publication of the Ameri... Quoraishi S, Mason G et. al.

Jun 29th, 2022 - To characterize the patterns of hearing loss and methods of hearing rehabilitation in the UK national cohort of adults with Alström syndrome. Retrospective review of electronic patient records. UK National multi-disciplinary team (MDT) Alström ser...

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...

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Clinicaltrials.gov  5 results

COhort for Bardet-Bield Syndrome and Alström Syndrome for Translational Research Monocentric Interventional Study
https://clinicaltrials.gov/ct2/show/NCT04461444

Sep 10th, 2021 - ALMS and BBS syndromes are rare diseases with overlapping features of multiple sensory and metabolic impairments, including diabetes mellitus. There are to date no specific treatments available and limited information on the natural history of the...

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...

Open-Label Rollover Study of PBI 4050 in Subjects With Alström Syndrome
https://clinicaltrials.gov/ct2/show/NCT03184584

Nov 3rd, 2020 - This is a Phase 2, open-label, single-arm, multi-centre study evaluating the long term safety and tolerability of PBI-4050 in subjects with Alström Syndrome who have completed the end-of-treatment (EoT) visit in a preceding ProMetic-sponsored Alst...

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...

Safety and Tolerability of PBI-4050 and Its Effects on the Biomarkers in Subjects With Alström Syndrome
https://clinicaltrials.gov/ct2/show/NCT02739217

Aug 29th, 2018 - This is a Phase 2, single-centre, single-arm, open-label study of the safety, tolerability, and effects on biomarkers of PBI-4050 in subjects with Alström syndrome. Approximately 18 subjects will be enrolled. The duration of study participation is...

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News  2 results

Pushback on Anti-Trans Policy; Imcivree's Longer Review; Good COVID & Diabetes News
https://www.medpagetoday.com/endocrinology/generalendocrinology/97379

Feb 27th, 2022 - Several medical organizations, including the Endocrine Society, are condemning the recent order by Texas Gov. Greg Abbott (R) directing the state's Department of Family and Protective Services to investigate instances of sex-change procedures in c...

$8B Price Tag for PCOS; Tymlos Label Update; Genetic Obesity Tx Seeks New Approvals
https://www.medpagetoday.com/endocrinology/generalendocrinology/94704

Sep 26th, 2021 - Eli Lilly is voluntarily recalling one lot of the glucagon emergency kit for low blood sugar with an expiration of April 2022 due to loss of potency, the FDA announced. The estimated cost to diagnose and treat women with polycystic ovary syndrome ...

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