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Connextion Property Management Rexburg – NIJMEGEN, The Netherlands – Monday, September 28, 2020: Khondrion, a clinical-stage biopharmaceutical company discovering and developing therapies targeting mitochondrial diseases, today announces that it has received rare pediatric disease (RPD) designation from the United States (US) Food and Drug Administration (FDA) for sonlicromonal for the treatment of patients with MELAS syndrome.
Sonlicromanol is Chondrion’s wholly-owned oral small molecule, potentially first-in-class and one of the most advanced disease-modifying drug treatments for emerging mitochondrial diseases. Currently in phase IIb clinical development, sonlicromanol has already received orphan drug designation for MELAS, Leigh disease, and patients with diabetes and maternally inherited deafness syndrome (MIDD) in Europe and for all inherited disorders of the mitochondrial respiratory chain in the US.
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The FDA grants RPD designation for serious and life-threatening diseases that primarily affect children 18 years of age or younger and fewer than 200,000 people in the United States. Subject to FDA approval of sonlicromanol for the treatment of MELAS syndrome, Khondrion may be granted a priority review voucher from the FDA, which may be redeemed to obtain priority review for any subsequent marketing application or may be sold or transferred to another company.
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We are pleased to have received this rare pediatric disease designation for our lead clinical stage development program. It signals the FDA’s recognition that MELAS syndrome presents a serious and unmet medical need and highlights the potential of sonlicromanol to become the first disease-modifying treatment for this devastating mitochondrial disease.”
A phase IIb study (KHENERGYZE) of sonlicromanol in MELAS spectrum disorders is ongoing in a number of internationally recognized mitochondrial disease centers in Europe. Khondrion plans to file a pre-IND (Investigational New Drug) application in the near term to further discuss the development plan for sonlicromanol in the US.
Khondrion is a private clinical-stage biopharmaceutical company that discovers and develops therapies targeting mitochondrial diseases. Founded by Prof. Jan Smeitink, a world leader in mitochondrial medicine, the company advances its proprietary science through a pipeline of wholly owned clinical and preclinical small molecule potential drugs.
The company’s in-house discovery engine uses unique live cell imaging technologies, patient-derived cell lines and predictive cell-based disease models to build a portfolio of promising compounds. Active discovery programs are developing new therapies, biomarkers, and new readout technologies in the field of mitochondrial diseases.
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To accelerate the discovery and development of its potential medicines for mitochondrial diseases, Khondrion collaborates with a global clinical and academic network and patient organizations internationally. Khondrion is based in Nijmegen, the Netherlands. For more information, visit www.khodrion.com
The syndrome of mitochondrial encephalopathy, lactic acidosis and stroke-like episodes belongs to a clinical spectrum of mitochondrial diseases most commonly caused by a mutation (m.3243A>G) of the MT-TL1 gene in the mitochondrial DNA (mtDNA). MELAS syndrome begins in childhood, usually between the ages of two and fifteen, with approximately 75% of cases reporting an onset of the disorder before the age of 20. Although rare, it is one of the most common types of mitochondrial disease caused by mtDNA mutations, with an estimated prevalence of MELAS spectrum disorders in adults and children at risk of 4.4 per 100,000 people. MELAS syndrome is a progressive and often fatal early-onset disorder that affects organs and tissues with a high energy demand, such as the brain and skeletal muscle. Current existing therapies only manage the symptoms and do not target the underlying cause of the disease.
Mitochondrial disease occurs when the mitochondria, which are found in all cells in the human body and are responsible for producing the energy needed by cells to function, are defective. This can lead to a wide range of serious and debilitating diseases that occur shortly after birth or later in life. Their signs and symptoms may include: cognitive problems, learning disabilities, blindness, deafness, heart failure, diabetes, fatigue, exercise intolerance, muscle weakness and problems walking, and growth retardation. Orphan diseases of the oxidative phosphorylation system such as Leigh disease, MELAS (mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes), spectrum disorders, MIDD (maternally inherited diabetes and deafness), LHON (Leber’s hereditary optic neuropathy ) and other respiratory chain oxidative phosphorylation disorders are all examples of mitochondrial diseases.
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