Monday, 28th February promotes the much needed awareness of rare diseases, generating change for the 300 million people worldwide living with a rare disease, their families and carers.
We are proud to work with amazing companies focused on developing new treatments for rare diseases.
Calliditas Therapeutics, which has received FDA approval to bring a new treatment for IgA Nephropathy to market.
Primary immunoglobulin A nephropathy (IgA nephropathy or IgAN or Berger’s Disease) is a rare, progressive, chronic autoimmune disease that attacks the kidneys, and can lead to progressive kidney damage and potentially a clinical course resulting in end-stage renal disease. TARPEYOTM, approved for the US, is the first and only FDA-approved treatment to reduce proteinuria in IgAN, to help these patients who are at risk of rapid disease progression.
Calliditas’ passion for its work is evident in its drive to educate. Its fantastic website dedicated to IgA Nephropathy is the place to learn more on this important work. IgA Nephropathy HCP Site | Calliditas Therapeutics (iganculprit.com)
We also commend the incredible work done by the IGAN Foundation on its commitment to research for a cure, public education and patient service.
We also want to acknowledge the work being done by [another] Swedish biotech Egetis Therapeutics. An orphan disease specialist, Egetis is making great strides in its Emcitate program, the first potential treatment for MCT8 deficiency, a rare genetic disease with high unmet medical need and no available treatment, affecting 1:70,000 males.
MCT8 deficiency is a rare and life-shortening genetic disease, causing serious consequences such as severely impaired neurocognitive development and motor function. In most of those affected, this results in an inability to maintain head control, sit independently and walk. In addition, MCT8 deficiency is associated with a state of chronic thyrotoxicosis manifested by severe underweight, muscle wasting and cardiovascular abnormalities. The disease only affects males, and the first symptoms usually emerge within the first months after birth. While rare, there is a universal need to improve the recognition, understanding and diagnosis of this devastating disease.
Egetis Therapeutics launched disease awareness initiatives in 2021, including the global Cuddly Toy Campaign. Last week, the #MCT8Hugs campaign was launched to drive better awareness of MCT8 deficiency among healthcare professionals and support diagnosis. www.mct8deficiency.com