PANTHERx Rare to exclusively distribute Kygevvi for TK2d
Global pharmaceutical company UCB has chosen PANTHERx Rare to distribute doxecitine and doxribtimine for the treatment of TK2d.
News
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Study identifies the most common G-tube problems
A recent study examined problems related to gastrostomy tubes (G-tubes), a type of feeding tube used by many patients with TK2d.
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Researchers examine screening for TK2 genetic variants
A newly developed lab assay for TK2d struggled to consistently detect the harmful effects of known disease-causing TK2 gene variants.
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Study highlights behavioral challenges in mitochondrial disorders
Neurobehavioral symptoms are common and significantly affect daily life in mitochondrial disorders, including TK2d.
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Study finds sildenafil improves outcomes in severe mitochondrial disease
Sildenafil improved symptoms and reduced metabolic crises in six patients with Leigh syndrome, a mitochondrial disease related to TK2d.
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Experimental drug PX578 boosts mitochondrial DNA in rare disorders
Experimental drug PX578 increased mitochondrial DNA in early laboratory studies of mitochondrial DNA depletion syndromes such as TK2d.
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Case study: First reported case of TK2d with neuropsychiatric symptoms
A recent case report describes a 59-year-old woman treated for psychiatric symptoms for over 30 years before being diagnosed with TK2d.
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Doxecitine and doxribtimine safe in patients with renal impairment
A study found that levels of doxecitine and doxribtimine were higher in those with renal impairment after dosing, but this was not tied to safety concerns.
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Public genome testing expands diagnoses for mitochondrial disease
Public genome sequencing identified a diagnosis in 20% of patients with suspected mitochondrial disease, including TK2d.
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New way to measure treatment success in primary mitochondrial disease
Goal attainment scaling may be a useful tool in clinical trials for primary mitochondrial diseases like TK2d.