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Rare Disease

Views & Analysis
The life-changing burden of my rare disease

The life-changing burden of my rare disease

I was first diagnosed with a rare autoimmune disease, Sjogren’s Syndrome, about ten years ago, after several years of medical struggles and misdiagnosis.

News
NICE1

Girl loses High Court battle over NICE review of BioMarin’s ...

News
Pfizer’s EU Vyndaqel okay ‘sets up $1.5bn sales next year’

Pfizer’s EU Vyndaqel okay ‘sets up $1.5bn sales next year’

News
eye

First UK patients get Novartis’ Luxturna gene therapy for bl...

News
Alnylam

Alnylam gets CHMP backing for second gene-silencing drug Giv...

News
Ipsen craters as pricey rare disease drug is put on hold again

Ipsen craters as pricey rare disease drug is put on hold aga...

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Editor's Picks

  1. NICE finally backs Enhertu for HER2-low breast cancer
  2. Novo Nordisk rebrands as Novo as new CEO charts turnaround
  3. MPs seek probe into UK-US pharma deal's impact on NHS
  4. FDA unveils framework for its expedited IND pilot
  5. WCLC: Lung cancer news from BioNTech, Akeso, and Roche
News
Lily gets FDA nod for SERD-CDK 4/6 combo in breast cancer

Lily gets FDA nod for SERD-CDK 4/6 combo in breast cancer

Eli Lilly's imlunestrant (Inluriyo) for breast cancer has received a new FDA approval, in combination with CDK4/6 inhibitor abemaciclib (Verzenio).

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Digital
Discover a new era of healthcare panel engagement
Sponsored

Discover a new era of healthcare panel engagement: Grounded ...

A new webinar from Konovo will address some of the critical topics impacting today’s healthcare market research industry.

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The rights of trademark owners are acknowledged. Website and content copyright © 2009-2026, pharmaphorum media limited or its licensors; all rights reserved. pharmaphorum – an EVERSANA company
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