This story was originally published on PharmaVoice. To receive daily news and insights, subscribe to our free daily PharmaVoice newsletter. Children with achondroplasia, the most common form of ...
With gene therapy, a cure for growth-hormone resistant dwarfism may be on the horizon. National University of Singapore, Yong Loo Lin School of Medicine Researchers from the NUS Yong Loo Lin School of ...
A novel drug for the most common cause of dwarfism accelerated children’s growth in a small but closely watched clinical trial, the company BridgeBio said Monday, advancing the latest treatment in ...
Oct 20 (Reuters) - The U.S. health regulator on Friday approved the expanded use of BioMarin Pharmaceutical's (BMRN.O), opens new tab once-daily injection to treat children under the age of 5 with the ...
A treatment for the most common cause of dwarfism met its goal of increasing height in a pivotal study, the drug’s maker said Monday, setting the stage for Food and Drug Administration approval. The ...
Sanofi has halted work on a phase 2 candidate for a rare growth disorder as part of a pipeline clear-out that saw the French pharma discontinue two other rare disease assets and one of its mRNA flu ...
A genetic disorder that slows bone growth and leads to dwarfism now has an approved drug, a biologic therapy developed by BioMarin Pharmaceutical. The European Commission decision announced Friday ...
The Food and Drug Administration (FDA) announced Friday that it had approved a drug for children with achondroplasia, the most common form of dwarfism. The drug, Voxzogo, is an injection to improve ...
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