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Acer Therapeutics

- PMLiVE

Bedrock are in the Top 3

Bedrock named No. 3 Young Agency by PMLive

Bedrock Healthcare Communications

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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

- PMLiVE

FDA rejects Sarepta’s new Duchenne drug over safety fears

FDA gave no warnings of concerns, says chief exec

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

Company to press ahead with phase 3 trial

- PMLiVE

Alexion’s Soliris adds new approval in rare disease NMOSD

First ever drug approved for rare autoimmune disease

- PMLiVE

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

- PMLiVE

Boehringer eyes rare disease use for respiratory blockbuster

Would help support respiratory franchise

- PMLiVE

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

- PMLiVE

Sobi shows strong growth despite haemophilia competition

Looking to M&A for continued growth

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