To her disbelief, Sanfillipo Syndrome, had symptoms that her daughter had been experiencing, which she thought were just ...

TD Cowen analyst Brendan Smith has maintained their bullish stance on DNLI stock, giving a Buy rating on November 1. Brendan Smith has given ...

REGENXBIO had positive updated long-term results from phase I/II/III CAMPSITE study using RGX-121 for the treatment of ...

SOUTH SAN FRANCISCO, Calif., Nov. 06, 2024 (GLOBE NEWSWIRE) -- (Nasdaq: DNLI) today reported financial results for the third quarter ended September 30, 2024, and provided business highlights. "As ...

A new study published in the journal of Molecular Psychiatry found that heparin medication was linked to a delayed diagnosis ...

DNLI reports a wider-than-expected loss in the third quarter of 2024. Nonetheless, the pipeline progress has been encouraging.

Children with Sanfilippo syndrome lack a single enzyme necessary to break down and recycle heparan sulfate, a large, complex molecule that is important for cell-cell interactions. When too much ...

Specifically, Sanfilippo makes the body unable to break down a molecule called heparan sulfate — a natural cellular substance usually metabolized and recycled through enzyme action. As a result ...

This work is an important contribution to understanding the role of FGF signaling in the induction of primitive-like cells in a 2D system of human gastrulation. The authors provide compelling evidence ...

HYOGO, Japan I October 31, 2024 I JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) announced the initiation of the first patient dosing in Japan in the Phase I ...

JR-441 is a proprietary recombinant heparan N-sulfatase capable of crossing the blood-brain barrier (BBB). MPS IIIA is a rare genetic disorder characterized by severe central nervous system (CNS ...