Cystinosis is an ultra-rare genetic metabolic disease that causes cystine an amino acid to accumulate in the body's organs.
It affects the kidneys, eyes, liver, muscles, pancreas, brain, and white blood cells, causing serious complications, including muscle wasting, growth delay, diabetes, hypothyroidism, and eventual kidney failure.
The disease has an estimated incidence of 1 to 2 per 150,000 births, with up to 100 Canadians thought to be affected TODAY.
Ocular manifestations of cystinosis can cause significant long-term morbidity and develop into more serious ocular complications, including blindness.
Left untreated, cystine crystal deposits form in the cornea, causing light sensitivity, eye pain, involuntary closing of the eyelids, and degeneration, resulting in partial or complete loss of vision.
Cystadrops (cysteamine ophthalmic solution, DIN 02485605) is a cysteamine solution approved in Canada for the treatment of corneal cystine crystal deposits in patients with cystinosis.
Recordati Rare Diseases Canada Inc. is a biopharmaceutical company committed to providing often overlooked orphan therapies to underserved rare disease communities in Canada.
Recordati Rare Diseases Canada Inc. is part of Recordati, an international pharmaceutical group committed to the research and development of new specialties with a focus on treatments for rare disease.
The company's Canadian corporate headquarters are located in Toronto, with global headquarters located in Milan, Italy.
European Medicines Agency recommends approval of Alvotech's biosimilar to Eylea
Gan & Lee presents trial results for novel diabetes therapies at ADA's 85th Scientific Sessions
TME Pharma and SERI partner to advance NOX-E36 in ophthalmology through option framework agreement
Innovent reports first participant dosed in new Phase 3 trial of mazdutide in China
Vivoryon Therapeutics presents positive meta-analysis data for varoglutamstat
Camurus and Lilly agree licensing deal for FluidCrystal incretins
Genprex signs new research agreement with University of Pittsburgh to advance diabetes gene therapy