Adam Pearson had the perfect response to a cruel troll who questioned whether he was going to the circus instead of the ...

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

She was born with achondroplasia, the most common form of short-limbed dwarfism. Those with this condition typically have an average-sized torso but shorter legs and arms. While the condition ...

The R&D head is looking for "clever science," which Friberg said is readily apparent in BioMarin’s recent research pact with CAMP4 Therapeutics. The partners are using CAMP4’s regulatory RNA platform ...

Gene regulation refers to the mechanisms that act to induce or repress the expression of a gene. These include structural and chemical changes to the genetic material, binding of proteins to ...

BioMarin's gene therapy pipeline has had setbacks, including a three-year delay in US approval of Roctavian and stalled development of PKU gene therapy BMN 307 as the Food and Drug Administration ...

Promising Daily Tablet Increases Growth in Children With Dwarfism Nov. 18, 2024 — A promising daily tablet is effective at increasing height and improving proportional limb growth in children ...

It seeks to understand the process of trait inheritance from parents to offspring, including the molecular structure and function of genes, gene behaviour in the context of a cell or organism (e.g ...

When BMRN reports fourth-quarter earnings, investors will likely focus on the sales performance of its dwarfism drug Voxzogo.

Peer ReviewDownload a summary of the editorial decision process including editorial decision letters, reviewer comments and author responses to feedback. Elevated cortisol in chronic stress and mood ...