NHS England’s Scorecard Denies Access to Treatment for Ultra Rare Diseases
Please sign and share our petition! Despite there being progress in the development of treatments for Ultra Rare Diseases, without a fair decision making process, access to these treatments will be denied.
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Why is this important?
Whilst new therapies are expensive for children and adults with ultra orphan diseases the number affected in England are usually in the tens and never total more than 500 affected individuals. These ultra rare diseases affect many organs of the body and usually result in death in childhood or early adulthood. Today even though the European Medicines Agency gave Marketing Approval for an Enzyme Replacement Therapy for children and young adults with MPSIVA, Morquio disease in April 2014 and the health departments in France, Germany, Austria, Italy and even Turkey are paying for Morquio sufferers to receive Enzyme Replacement Therapy, 77 children and adults in England are denied Enzyme Replacement Therapy and to be treated at home.
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