The skeleton undergoes continuous remodeling throughout life in response to diverse environmental stimuli. Osteoclasts, which are specialized cells on the bone surface, break down old bone tissue (in a process known as bone resorption) and build it back up. Dysregulation of osteoclast formation and function can lead to bone fragility, including osteoporosis, which is estimated to affect over 10 million people globally.
As expected, the FDA granted an approval for Amondys 45 (casimersen), a new Duchenne muscular dystrophy (DMD) therapy developed by Sarepta Therapeutics Inc.
Australian researchers at the Walter and Eliza Hall Institute of Medical Research in Melbourne have developed new yeast- and antibody-based methods to determine how tryptophan C-mannosylation, an unusual protein glycosylation modification, impacts the stability and function of disease-relevant proteins.
In the wake of Study 045’s failure with Translarna (ataluren) in nonsense mutation Duchenne muscular dystrophy, PTC Therapeutics Inc. is “trying to thread the needle between the notion of getting Study 041 completed in the third quarter of 2022 vs. getting accelerated approval now,” CEO Stuart Peltz said.
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