The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie ...

Endothelial stiffening induced by Western diet was proposed to be an important factor in vascular dysfunction. In this study, we determine the role of endothelial CD36 (cluster of differentiation 36) ...

In March 2023, Tevard announced a four-year global research collaboration with Vertex Pharmaceuticals to develop novel tRNA-based therapies for Duchenne muscular dystrophy (DMD) caused by nonsense ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, shared the following update related to ELEVIDYS ...

OCT parameters included central macular thickness (CMT), subfoveal choroidal thickness (SFCT) and retinal and choroidal thickness at site of CNV. Patients were treated with anti-vascular endothelial ...

The deal included participation from Braidwell LP, Cormorant Asset Management, Driehaus Capital Management, Invus, Janus Henderson Investors, MPM BioImpact, OrbiMed, Paradigm BioCapital Advisors ...

New therapeutic targets are emerging for childhood visual disorders, although the safety and efficacy of novel therapies for diseases such as ROP or retinal dystrophies are not yet clear.

Plaques are also detected in the striatum and cerebellum. Cerebral amyloid angiopathy occurs in cortex, hippocampus, striatum, and cerebellum. Plaque-associated neuritic dystrophies are observed ...

CAMBRIDGE, Mass., March 31, 2025--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, granted equity awards on March 31, 2025 ...

Aurion will operate as a separate company with full support from Alcon to advance its clinical-stage allogeneic cell therapy asset, AURN001, into Phase 3 for corneal edema secondary to corneal ...