Tag: Metabolic disease

Biotinidase deficiency, a rare inherited metabolic disorder, is detected in approximately 1 in 61,067 newborns worldwide through mandatory screening programs. This condition, if left untreated, can cause severe neurological and dermatological issues, making it critical to understand **what medication is used for biotinidase** to prevent serious complications.

First approved by the FDA in 2002 for hereditary tyrosinemia type 1 (HT-1), nitisinone revolutionized the treatment of this life-threatening genetic disorder. But **which enzyme does nitisinone block** to achieve such a dramatic clinical outcome, and how does this mechanism prevent severe organ damage?

According to the National Institutes of Health, a plasma free carnitine concentration of 20 mcmol/L or less is considered abnormally low. Understanding **what can cause carnitine deficiency** is crucial for proper diagnosis, as it can stem from genetic defects, acquired medical conditions, or medication side effects.

Based on recent clinical trial findings, Efpeglenatide, an investigational glucagon-like peptide-1 receptor agonist (GLP-1 RA), has demonstrated significant improvements in glycemic control and body weight loss for patients with type 2 diabetes (T2D). The drug’s potential indications extend beyond blood sugar management, showing promising results for reducing cardiovascular and renal risks in high-risk patient populations.

As of September 2025, the FDA has already approved 29 novel drugs this year, signaling a robust pipeline of innovation [1.2.1]. The landscape of medicine is constantly evolving, so the key question for patients and providers alike is: what new drugs are coming out?

Estrogen-related receptors (ERRs) are central regulators of energy metabolism, and recent studies on synthetic ERR agonists like SLU-PP-332 show they can mimic the metabolic benefits of exercise [1.5.3, 1.6.2]. So, what is an ERR agonist? It's a molecule that activates these powerful receptors.