Sarah Neidler,  —

Rare Disease Day 2023

Recent Posts

• New MFSD8 mutation identified in girl with CLN7 disease
• Use of gemfibrozil shows promise in mouse model of juvenile Batten
• Doctors are trained to think horses, but what about the zebras?
• Grant program supports research projects Down Under
• Cellular storage material may track CLN2 disease therapy efficacy
• Regenxbio details upcoming RGX-381 trial for CLN2 vision loss
• Loss of visual acuity, quality may be first sign of CLN3 disease
• Honoring my sister, who taught me how to persevere
• Problems in retinal nourishing cells may drive juvenile Batten vision loss
• Neurogene discontinues research program for CLN7 Batten