Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before birth. Scientists at St. Jude Children's Research Hospital led the first in utero treatment of SMA ...

The phase 1b trial will enrol patients with geographic atrophy secondary to age-related macular degeneration (AMD), a leading cause of blindness. While there are multiple therapies available for ...

Astellas has shored up the competitive profile of Izervay as a treatment for eye disorder geographic atrophy (GA), getting FDA approval for an extended treatment duration. The US regulator turned ...

Researchers assessed the association between best corrected visual acuity and ellipsoid zone parameters at baseline in eyes with geographic atrophy. Ellipsoid zone preservation is a key structural ...

In the month of March, not only did trials progress, but approvals for new and expanded uses of treatments were issued.

Michael L. Krychman, MD; Jan L. Shifren, MD, NCMP; James H. Liu, MD; Sheryl L. Kingsberg, PhD; Wulf H. Utian, MB BCh, PhD, DSc(Med), FRCOG, FACOG, FICS ...

In Krabbe disease, galactocerebroside accumulates in the peripheral and central nervous system producing cerebral atrophy, loss of myelin, gliosis and globoid cells.7 There are three forms of Krabbe ...