The FDA has delayed a decision on Ascendis Pharma’s dwarfism drug candidate TransCon CNP, pushing back the deadline by three months to buy time to review recently submitted information.

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 ...

If approved, Ascendis’ TransCon CNP would become the second therapy for achondroplasia, challenging BioMarin’s Voxzogo.

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 ...

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 ...

At the Rea home in Newark, you'll likely find Christopher sitting in the family room, killing monsters in one of his many video games. His little sisters, Ashlyn and Ember, will be running around him, ...

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 ...

Rare growth disorders are a diverse group of uncommon conditions that affect an individual’s physical development. They may stem from genetic mutations, hormonal imbalances, or other complex factors.