Rare Disease Day 2015

Sobi shares study findings on HT-1 adherence in adolescent patients, to highlight life-long health outcomes for rare disease patients - Seite 2

Sobi is building on the findings and is actively involved in the collaborative development of tools that aim to address the adherence challenges identified in the research. The first part of the outcomes of the adherence programme has been developed in the United Kingdom and will be introduced to the broader HT-1 healthcare community during the course of 2015.

Sobi is also supporting activities in connection with Rare Disease Day in several countries:

Austria: Sobi is supporting the nationwide Rare Disease Day campaign launched on 27 February 2015 in the Austrian Parliament, led by the Minister of Health. The aim of the event is to raise awareness of rare diseases in the Parliament.

Germany: Sobi organised a charity run and walk on 25 February 2015 during  the 59^th Annual Meeting of the Society of Thrombosis and Hemostasis Research, the largest German-speaking haemostasis congress. Sobi will donate up to €25 for each participant in the event, with the objective of raising funds to continue to support the work of the representative organisations in the field of haemophilia. The donation will, in equal parts, go to the German Haemophilia Society (DHG) and Interest Group of Haemophiliacs (IGH).

North America: Sobi will engage in, and support, several events organised by the Rare Disease Legislative Advocates taking place between 23-27 February 2015.

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About HT-1
Hereditary Tyrosinaemia type 1 (HT-1), is a rare genetic disorder, which can cause liver failure, kidney dysfunction and neurological problems. About one newborn child in 100,000 has the disorder, although geographical variations exist.Tyrosine is one of 20 amino acids present in all proteins. Excess tyrosine is degraded in several steps, but in HT-1, one of the enzymes in this degradation, fumarylacetoacetat hydrolase (FAH), is deficient. Tyrosine and its toxic metabolites thus build up in the body and cause serious medical problems.

About Rare Disease Day
Rare Disease Day takes place on the last day of February each year. The main objective of Rare Disease Day is to raise awareness amongst the general public and decision-makers about rare diseases and their impact on patients' lives. Rare Disease Day was first launched by EURORDIS and its Council of National Alliances in 2008. The theme for Rare Disease Day 2015 is "Living with a Rare Disease" with the slogan "day-by-day, hand-in-hand". The focus is on the daily lives of patients, families and caregivers who are living with a rare disease and it pays tribute to the millions of parents, siblings, grandparents, spouses, aunts, uncles, cousins, and friends whose daily lives are impacted and who are living day-by-day, hand-in-hand with rare disease patients.www.rarediseaseday.org