A group of rare disease charities specialising in acquired and inherited rare bone marrow failures have come together to form a collaborative alliance – Together for Heathy Marrow. The charities include Fanconi Hope, DC Action, PNH Support, DBA UK and the Aplastic Anaemia Trust. The alliance has been initiated in recognition that the operating environment
by Dan StantonThursday, April 11, 2019 12:04 pm Russian biopharma Generium has launched Elizaria, a biosimilar version of Alexion’s Soliris (eculizumab) developed using the SUREtechnology Platform from Selexis. Approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), Soliris (eculizumab) pulled in $3.6 billion (€3.2 billion) in global sales for Alexion Pharmaceuticals in 2018.
See the latest edition of Rare Revolution magazine for Suzie’s story about life with PNH (page 28). https://edition.pagesuite-professional.co.uk/html5/reader/production/default.aspx?pubname=&edid=5e15642b-f320-410c-afd5-200f9f8f7b1e
This blog was written for the Protect ERNs website. Please sign up here to support the campaign. PRESERVATION OF THE UK IN ERNs – WHAT COLLABORATION MEANS FOR PATIENTS AFFECTED BY RARE BLOOD DISORDERS Maria Piggin, Chair of PNH Support, and Sophie Wintrich, CEO of MDS UK Maria Piggin and Sophie Wintrich hold voluntary positions as European
“This approval is based on comprehensive results from two Phase 3 studies, which were recently published in Blood.In these studies, which included 441 patients who had either never been treated with a complement inhibitor before, or who had been stable on SOLIRIS, the efficacy of ULTOMIRIS administered every eight weeks was non-inferior to the efficacy
For the last two years PNH Support has printed Christmas Cards using artwork generously donated by a patient’s sister. We have regrettably decided not to continue to print these in future however we still have Christmas cards left over from the last 2 years if anyone would like to buy some (pictures above of 2
Genetic Alliance UK still have spaces available in upcoming focus groups for their research project CONCORD (CoOrdiNated Care Of Rare Disease). They are currently recruiting for focus groups for rare disease patients, carers and healthcare professionals. These focus groups are crucial to help the researchers understand the needs and concerns of both patients and healthcare