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hypophosphatasia infantile hypophosphatasia+1 moreTrials & approvals

The investigational enzyme replacement therapy efzimfotase alfa, developed by AstraZeneca, failed to meet its primary endpoint in a phase III trial for hypophosphatasia, showing no improvement in the 6-Minute Walk Test. Additionally, a study suggests a link between polyendocrine metabolic ovarian syndrome and increased rates of dementia and Alzheimer's disease in women.

Dementia and Alzheimer's disease may be more common in women with polyendocrine metabolic ovarian syndrome (PMOS), formerly known as polycystic ovary syndrome (PCOS), an association likely driven by metabolic comorbidities. (Journal of the Endocrine Society) The investigational enzyme replacement therapy efzimfotase alfa failed to hit its primary endpoint of improvement in the 6-Minute Walk Test in a phase III trial of treatment... Dementia and Alzheimer's disease may be more common in women with polyendocrine metabolic ovarian syndrome (PMOS), formerly known as polycystic ovary syndrome (PCOS), an association likely driven by metabolic comorbidities. (Journal of the Endocrine Society) The investigational enzyme replacement therapy efzimfotase alfa failed to hit its primary endpoint of improvement in the 6-Minute Walk Test in a phase III trial of treatment-naive patients with hypophosphatasia, according to maker AstraZeneca. A low-fat plant-based diet was associated with improved liver fat content, insulin sensitivity, and beta-cell function in adults with type 2 diabetes in a single-arm trial. (BMJ Open Diabetes Research & Care) From 2017 to 2023, presentation rates for nonbinary youth increased 2.7-fold compared with binary transgender youth at an Australian pediatric gender clinic. Zealand Pharma said its investigational amylin analog petrelintide led to an average weight reduction of up to 9.2% at 28 weeks and with low discontinuation rates, compared with a 2% weight reduction with placebo, in a phase II trial of adults with overweight/obesity and type 2 diabetes. News and commentary from the endocrinology world

Treatment & Clinical News22 days agoRead at source →

Original title: “Bootleg Retatrutide Deaths? PMOS and Dementia; Weight-Loss Drug to Be Discontinued | MedPage Today”

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A systematic review highlights the prevalence of hypophosphatasia across general and specific patient populations. This research provides valuable insights into the disease's occurrence, which may inform future studies and treatment approaches.

A study published in PubMed highlights the diagnostic yield of laboratory-based screening for hypophosphatasia in Chinese children presenting with low alkaline phosphatase levels. This research could enhance early detection and management of this rare bone disorder.

A case report details an adult with hypophosphatasia carrying a single heterozygous mutation in the ALPL gene. This finding contributes to the understanding of genetic variations associated with the disease.

Researchers have developed new knock-in mouse models for hypophosphatasia, providing valuable tools for studying the disease's mechanisms and potential therapies. These models may enhance understanding of the genetic underpinnings of hypophosphatasia.

A recent study highlights the imbalance of osteoglycin and sclerostin as potential bone-derived markers in hypophosphatasia, suggesting systemic involvement in the disease. This research could pave the way for new diagnostic and therapeutic strategies.

A recent study highlights the significance of low alkaline phosphatase levels in adults as a potential indicator for diagnosing hypophosphatasia during osteoporosis evaluations. This research could enhance diagnostic accuracy for a rare bone disorder.

A study reports a 17-year follow-up of middle-aged siblings diagnosed with hypophosphatasia, revealing a novel intronic and a rare missense mutation in the ALPL gene. This research contributes to the understanding of genetic factors in hypophosphatasia.

A new study presents an integrated genetic and biochemical approach for diagnosing patients with bone disorders characterized by low alkaline phosphatase levels. This research could enhance understanding and treatment strategies for these rare conditions.

A recent study investigates the impact of comorbid chronic kidney disease on late-onset hypophosphatasia in mice treated with asfotase alfa. The findings contribute to understanding the complexities of managing this rare disease in patients with additional health conditions.

A case report highlights the bilateral healing of pseudofractures in a patient with hypophosphatasia treated with teriparatide. This finding suggests potential therapeutic benefits of teriparatide for managing bone complications in this rare disease.

A study validates proposed diagnostic criteria for hypophosphatasia in adults using a Spanish cohort. This research enhances the understanding and identification of this rare bone disease.

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