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 ...

How is the size of an animal reduced over a series of generations? In other words, what is the process by which, for example, ...

Aubrey Smalls was taking a photo of a suitcase he was considering purchasing, when he noticed his camera moving Courtesy of Aubrey Smalls Aubrey Smalls was in TJ Maxx when he noticed his camera ...

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 ...

The company raised $200 million after early findings hinted its therapy could be more potent than others like it, including Sarepta Therapeutics’ Elevidys.

Gene expression is the generation of a functional gene product from the information encoded by a gene, through the processes of transcription and translation. Gene products are often proteins ...

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 ...

But when he turned around, he was shocked to see two strangers picking him up. "People just feel like they can touch us, and that goes on such a wide spectrum," Smalls, who was born with dwarfism, ...