Winst gevend schreef op 13 oktober 2025 16:12:
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Ook KL 1333 hangt aan een zijden draadje.Elamipretide, too, was rejected twice,
and only greenlit after more than a year of back and forth with the manufacturer.
Still, mitochondrial disease researchers hope they are finally on a roll.
FDA is currently considering another drug,
MT1621, for an extremely rare mitochondrial disease
known as thymidine kinase 2 deficiency (TK2d).
“I’d be floored if it wasn’t approved,” Koenig says.
At least seven other treatments for mitochondria-related illnesses are in clinical trials,
and academic researchers and a host of new biotechs are pursuing more potential therapies. “There is an explosion of studies,” says geneticist and clinical researcher
Fernando Scaglia of Baylor College of Medicine. “We are excited.”
Dan lijkt het KL 1333 Pharming studie resultaat jaar in 2029, nu al niet meer relevant.Gelukkig hebben ze nog 150 joenja klanten. :-)
8 oktober 2025:
www.science.org/content/article/first...