Rare Disease

Views & Analysis

engineering and genetic editing through the Crispr technique. DNA chain breaking down. 3D rendering

Gene addition – a new chapter in treating rare diseases

The scientific advancements of the past 30 years have transformed our understanding of the potential of gene therapy and how it may be used to tackle rare diseases.

Views & Analysis

Increasing patient involvement in research to tackle unmet n...

It could be said that there is no greater unmet need than a complete and absolute absence of treatment options.

Views & Analysis

Dutch biotech Pharming plans to challenge big pharma in rare...

The introduction of Shire’s preventive treatment for hereditary angioedema has shaken up the market for drugs for this rare disease.

Views & Analysis

Rare Youth Project: Can you empower the next generation of r...

The next generation of rare disease advocates need a little support in getting their voices heard as part of the Rare Youth Project.

Views & Analysis

Patient centricity is about delivering with, not for, patien...

Patient centricity starts with communication and collaboration and these are the only ways to ensure people living with rare diseases like scleroderma will be able to access potentially transfo

Views & Analysis

Balancing expectations: An unmet need in the orphan disease ...

There are between 6000 and 8000 rare diseases in the world, and one in ten people in Europe and the US have a rare disease; 75% of rare diseases affect children, and 30% of rare disease patients di

Gene addition – a new chapter in treating rare diseases

Increasing patient involvement in research to tackle unmet n...

Dutch biotech Pharming plans to challenge big pharma in rare...

Rare Youth Project: Can you empower the next generation of r...

Patient centricity is about delivering with, not for, patien...

Balancing expectations: An unmet need in the orphan disease ...

The long-delayed promise of the microbiome, with Sam Possemi...

How to meet your physicians where they are online

Rare Disease

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