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