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.
vTv Therapeutics secures USD51m private placement for cadisegliatin Phase 3 study
AbbVie names new chief executive officer
SkinBioTherapeutics expands foot care products in AMEA with Dermatonics partnership
Diamyd Medical granted US FDA Fast Track designation for Diamyd diabetes treatment
Insulet's Omnipod 5 integration with Abbott FreeStyle Libre 2 Plus Sensor secures CE Mark approval
Kyowa Kirin enrols first subject in phase two tivozanib eye drops (KHK4951) clinical trial
China accepts GSK's Shingrix application for at-risk adults
Novo Nordisk to boost manufacturing capacity with acquisition of three fill-finish sites
Breye Therapeutics commences phase 1b/2a danegaptide clinical trial
DexCom Inc breaks ground on new manufacturing facility in Ireland
VibroSense Dynamics AB secures provisional patent for chemotherapy-induced nerve damage prediction
Innovent Biologics signs collaboration agreement with Sanegene Bio USA
Japan approves GSK's filing for Arexvy vaccination for at-risk adults