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European legislation must be revised to boost innovation in rare disease

This article has been developed and funded by Chiesi Group Rare Diseases.  Inflation and rising public debt are creating barriers for the rare disease community to access the latest therapies. Public healthcare budgets are being reduced, making access to new treatments difficult while stricter evidence requirements further limit availability across Europe. The lack of treatment … Continued
Rare Diseases Q3 2024

Free genomic sequencing offers hope for children living with a rare disease

There are 350 million undiagnosed children worldwide, with most lacking access to genomic testing for proper diagnosis and care options. In a world where medical advancements often seem reserved for the fortunate few, iHope Genetic Health (iGH) is striving to change the narrative for children living with rare diseases. Call for equitable genetic testing in … Continued
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How far are we from being able to treat mitochondrial disorders?

This page has been funded by UCB, but editorial control lies with the contributors. UCB only performed a medical accuracy review but was not otherwise involved in the content. Mitochondrial disorders are a group of rare diseases that affect 1 in 5,000 live births. They can present at any age and have a drastic impact … Continued