Ledaga recommended by NICE for rare type of lymphoma pharmatimes.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from pharmatimes.com Daily Mail and Mail on Sunday newspapers.
:
The rising prevalence of urea cycle disorders is expected to increase the demand for treatment options for urea cycle disorders (UCD), which is expected to drive the growth of global urea cycle disorders treatment market during the forecast period. According to the Urea Cycle Disorders Consortium (UCDC), a part of the Rare Diseases Clinical Research Network (RDCRN), combined prevalence of urea cycle disorders is around 1 in 30,000 people in the U.S in 2017
Among regions, North America is expected to hold dominant position in the global urea cycle disorders treatment market during the forecast period, owing to key companies focusing on product launches for the treatment of Urea cycle disorders. For instance, in January 2021, Recordati Rare Diseases Inc. received the U.S. Food and Drug Administration (FDA) approval for CARBAGLU for the treatment of acute hyperammonemia.
SMC endorses Alexion’s Ultomiris
11th May 2021
The Scottish Medicines Consortium (SMC) has accepted a treatment for an ultra-rare disease that can cause progressive injury to vital organs via damage to the walls of blood vessels and blood clots in its May decisions.
Restricted use of Alexion’s Ultomiris (ravulizumab) has been recommended by the SMC, for the treatment of patients with a body weight of 10kg or above with atypical haemolytic uremic syndrome (aHUS).
Patients in Scotland who are complement inhibitor treatment-naïve or have previously received Alexion’s Soliris (eculizumab) for at least three months, and who have evidence of response to Soliris, are now eligible for the new treatment.