For over 40 years Masters has supported healthcare professionals and their patients arounds the world; providing access to life-saving medicines for rare diseases.
There are over 7,000 recognised rare diseases but approximately only 500 approved treatments. An estimated 300 million people around the world suffer with a rare disease,but prevalence in each disorder is low and medical expertise is rare. As such, it can take a long time for patients to receive a diagnosis and the effective treatment to help improve their quality of life.
Our impact in rare disease
UK
Sickle Cell Disease
In partnership with Theravia® we are the exclusive distributor for Siklos® in the UK. Siklos® is indicated for the treatment of vaso-occlusive crises in patients with sickle cell disease.
BRAZIL
Sickle Cell Disease
Masters is the marketing authorisation holder for Siklos® in Brazil, which is now approved by ANVUISA for the treatment of vaso-occlusive crises in patients with Sickle Cell Disease.
Spinal Muscular Atrophy
Early access supply of Spinraza® (Nusinersen) in partnership with Biogen in Brazil for patients with spinal muscular atrophy.
Multiple Myeloma
Early access supply of Revlimid® (lenalidomide) in partnership with Celgene Brazil for patients with multiple myeloma.
USA
Duchenne’s Muscular Dystrophy
Supplied Emflaza® (Deflazacort) to patients with Duchenne’s Muscular Dystrophy through named patient supply and early access programmes. Masters partnered with patient advocacy groups and Parent Project Muscular Dystrophy (PPMD).
CAYMAN ISLANDS
LG-UTUC
In partnership with Uroge, Masters delivered training to healthcare professionals on Jelmyto® (mitomycin) and supplied the treatment to the Cayman Islands Health Services Authority (CIHSA) for patients with low-grade Upper Tract Urothelial Cancer (LG-UTUC).
Haemophilia B
We work in partnership with the CIHSA to provide Alprolix® (coagulation factor IX) for patients suffering with haemophilia B across the Cayman Islands.
WORLDWIDE
LHON
Early access supply of Raxone® (Idebenone) globally for the treatment of visual impairment in adolescent and adult patients with Leber's Hereditary Optic Neuropathy (LHON).
Paroxysmal Nocturnal Haemoglobinuria (PNH)
Supplied healthcare providers worldwide with Spinraza® (eculizumab) for the treatment of ambulatory patients.
IRAQ
Wilson’s Disease
We supply Coagudex® (Human coagulation factor X 250IU) through an early access programme for patients with hereditary factor X deficiency.
We partnered with Univar to distribute Cufence® (Trientine Hydrochloride) to patients in Iraq with Wilson’s disease. Cufence® through early access.
TRINIDAD & TOBAGO
Thalassaemia
Annual supply of Ferriprox® (deferiprone) through NIPDEC national tender for the treatment of Thalassaemia.
BERMUDA
Haemophilia A
We work in partnership with King Edwards Memorial Hospital (KEMH) to supply Elocta® (efmoroctocog alfa) to treat patients with Haemophila A.
Sickle Cell Disease
Sickle cell disease (SCD) is a genetic blood disorder that
affects around 8 million people worldwide. It is most
commonly present in those of African and Caribbean
origins. The condition is marked by the presence of
abnormal haemoglobin, known as haemoglobin S, which
causes red blood cells to take on a rigid, sickle shape.
These sickle-shaped cells can lead to a range of serious
health problems affecting every aspect of a person’s life.
Siklos®
We are proud to partner with Theravia® to bring Siklos® to sickle cell patients in the UK and Brazil.
We are the exclusive distributor for Siklos® in the UK, working with the NHS to provide more treatment options for patients.
Access information about Siklos® here
To find out more about Siklos® in Brazil visit https://masterspharma.com.br/
