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- PMLiVE

Spectrum Pharmaceuticals

Life with multiple myeloma – why I’m a lucky man living the dream

Multiple myeloma patient Bob Munro explains what it's like to live with the rare blood cancer and how his diagnosis led him on the journey of a lifetime – cycling...

Blue Latitude Health

- 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

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

- PMLiVE

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

Orphan drugs market to soar to $224bn by 2024

Cell and gene therapies to fuel growth

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