PTC Therapeutics has been granted conditional approval from the European Commission for its Duchenne muscular dystrophy (DMD) drug Translarna (ataluren), which has yet to produce Phase III data.
PTC Therapeutics has been granted conditional approval from the European Commission for its Duchenne muscular dystrophy (DMD) drug Translarna (ataluren), which has yet to produce Phase III data.
Translarna has been approved to treat nonsense mutation Duchenne muscular dystrophy in 28 EU countries, as well as Iceland, Liechtenstein...
A new muscle-building front is opening in the fight against obesity, with Lilly among the leaders. But it plans to remain tight-lipped on upcoming Phase II data for its myostatin inhibitor in combination with Novo’s Wegovy for fear of playing into the hands of its rival.
The Mereo-partnered drug is in pivotal development for osteogenesis imperfecta. Ultragenyx also recently reported positive Phase III data for a rare disease gene therapy.
The company sees a $5bn opportunity to treat children with achondroplasia and the less severe growth disorder variant, hypochondroplasia.
Phase I/II data for its ADC candidates in myotonic dystrophy type 1 and Duchenne muscular dystrophy indicate potential for biomarker-based accelerated approval in both diseases, Dyne asserts.
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Pipelines in bioconjugates continue to develop, from ADCs to XDCs, with radionuclide drug conjugates (RDCs) and antibody-oligonucleotide conjugates (AOCs) leading the expansion.
Interim results from the Phase III REMARK study presented at WCLC showed the Chinese company’s second-generation ALK/ROS1 inhibitor met its primary endpoint as a first-line therapy in ALK-positive advanced NSCLC.
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