ALLMedicine™ Alström Syndrome Center

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...

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,|

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...

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...

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...

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...

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...

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...

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...

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...

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...

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 ...