Parkinson’s disease (PD) is a neurodegenerative disorder. But not just. And it may not start that way.
There is increasing evidence that a-synuclein, the protein whose aggregates eventually destroy midbrain dopaminergic neurons in PD (and that are the cause of other diseases collectively known as the synucleinopathies), first aggregates “in enteric neurons, the neurons that control gastrointestinal function,” Collin Challis told BioWorld.
Ultragenyx Pharmaceutical Inc.’s top-line win in January with DTX-301 gene therapy in ornithine transcarbamylase (OTC) deficiency seemed to presage even better things to come later this year, and analysts more recently hailed fourth-quarter earnings that showed satisfying progress with Crysvita (burosumab).
Spain's Sanifit Laboratoris SL, a company developing treatments for calcification disorders, has dosed the first patient in a phase III trial of its lead asset, SNF-472, for the treatment of the rare and sometimes deadly disease calciphylaxis, a calcium accumulation disorder.