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Tapping the potential of rare diseases: A journey into uncharted territory

Delivering effective treatment for rare diseases remains a major unmet medical need. Fortunately, scientific advances are rapidly enhancing our ability to identify, diagnose and ultimately treat these conditions. Coupled with...

Porterhouse Medical Group

Article: Engaging patient advocacy groups

The key to conducting effective rare disease research

Research Partnership

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Rivals rise as safety issue mars Pfizer’s DMD gene therapy trial

Company to press ahead with phase 3 trial

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Alexion’s Soliris adds new approval in rare disease NMOSD

First ever drug approved for rare autoimmune disease

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Acer Therapeutics hit by FDA filing rejection

Shares plunge as regulator calls for new trial

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NHS England wants money-back guarantee on Orkambi

Vertex resisting real-world data proposal

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Boehringer eyes rare disease use for respiratory blockbuster

Would help support respiratory franchise

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Speedy approval for Pfizer’s Onpattro rival

Approved in two different formulations

Biomarin

EU nod for BioMarin’s second PKU therapy Palynziq

Palyniq is significant, but gene therapy pipeline most anticipated

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Sobi shows strong growth despite haemophilia competition

Looking to M&A for continued growth

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Unruffled by ‘outlier’ claims, Vertex CEO upbeat on Orkambi breakthrough

NHS England going public with price makes deal impossible, says Leiden

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‘Inflexible’ NICE blocking access to rare disease drugs

Spinraza most high profile example of access problem

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