Hypoketotic Hypoglycemia Explained in Simple Terms Hypoketotic hypoglycemia is a medical condition characterized by low blood sugar levels, with a focus on fatty acid oxidation disorders. It occurs when the body is unable to efficiently break down fats as an energy source, leading to a buildup of toxic by-products and [...]
Ultragenyx Announces U.S. FDA Approval of Dojolvi™ (UX007/triheptanoin), the First FDA-Approved Therapy for the Treatment of Long-chain Fatty Acid Oxidation Disorders Fourth U.S. FDA Approval for Ultragenyx in the Past Three Years – All for Rare Diseases which Previously Had No Approved Therapies Ultragenyx to Host Conference Call Today at 4:00 pm [...]
INFORM Conference Explores Future of Disorders of Fatty Acid Oxidation International Network for Fatty Acid Oxidation Research and Management showcased advancements, next-gen researchers Global experts, practitioners, researchers and family members gathered this fall for the ninth meeting of the International Network for Fatty Acid Oxidation Research and Management. Held in [...]
How is CPT2 Diagnosed? Most people never think about the adverse impacts of dieting or skipping a meal on a busy day. We assume that this is a normal function of the body that can be handled by the body. Under normal circumstances, this is true as the body can [...]
LCFAOD Symptoms Linked to Alterations in Complex Lipids. Accumulated altered versions of complex lipids sphingomyelins, ceramides, and cardiolipin are likely responsible for LCFAO symptoms according to a study. A recently published hypothesis article by the International Journal of Molecular Medicine indicates that retinopathy and neurodegenerative symptoms associated with patients that [...]
Efficacy of a Diet Supplemented With Triheptanoin Proven Greater Than IMB-203 in Treating VLCAD Deficiency Triheptanoin (C7) has shown effectiveness in treating CoA dehydrogenase VLCAD deficiency, a type of LCFAOD. It has also shown to have greater efficacy than IMB-203. The study found that C7 reduced hepatic steatosis, whereas IMB-203 [...]
LCFAOD Symptoms Linked to Alterations in Complex Lipids. Accumulated altered versions of complex lipids sphingomyelins, ceramides, and cardiolipin are likely responsible for LCFAO symptoms according to a study. A recently published hypothesis article by the International Journal of Molecular Medicine indicates that retinopathy and neurodegenerative symptoms associated with patients that [...]
Ultragenyx CEO and Founder Discusses Fundamental Flaws in Drug Development Process Emil Kakkis, MD, Ph.D., founder and CEO of Ultragenyx sat down for an interview with Larry Luxner, senior correspondent for Rare Disease Advisor. Dr. Kakkis spoke about the inefficiency and often lack of effectiveness of the current development paradigm. [...]
New Study Finds C7 Could be Effective Against VLCAD According to a recent study by Xue-Jun Zhao, MD, of Children’s Hospital of Pittsburgh in Pennsylvania, treatment with triheptanoin, also known as C7, could be effective in combating VLCAD deficiency. Dr. Zhao’s lecture was presented as part of the International Network for Fatty Acid [...]
Study Helps Determine the Best Dosing Frequency of Triheptanoin for the Treatment of LCFAOD A recent study by Wendy Putnam, PhD, from Ultragenyx developed a new model that could accurately estimate the elimination half-life of triheptanoin in patients with LCFAOD. This model can be used to calculate the best dosing frequency of triheptanoin marketed under [...]
