Cystinosis

The American Kidney Fund has launched the Rare Kidney Disease Action Network, an advocacy initiative for patients with rare conditions, according to a press release.

For years, cystinosis — the rare, inherited and life-long condition caused by abnormal buildup of the amino acid cystine in the body — was considered a fatal pediatric disease.

WAILEA, Hawaii — In this Healio Video Perspective from Hawaiian Eye 2024, Cynthia Matossian, MD, FACS, discusses how a soft contact lens may deliver medication to the front of the eye as an alternative to eye drops.

This year, the national virtual camp hosted by the American Kidney Fund will include a program specifically for children with cystinosis.

As a nephrologist at Montefiore Medical Center/Albert Einstein School of Medicine, I have seen several young patients with rare diseases come into my clinic.

The FDA has approved Cystadrops, a viscous eye drop solution that reduces corneal cystine crystal deposits in patients with cystinosis, according to a press release from Recordati Rare Diseases.