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Rare diseases: Novartis’ Fabhalta recommended by CHMP for C3 glomerulopathy

Approximately one to two people per million worldwide are diagnosed with the ultra-rare kidney disease every year

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Rare diseases: CHMP recommends Krystal’s Vyjuvek for dystrophic epidermolysis bullosa

There is currently only one treatment authorised for the ultra-rare genetic blistering disease

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Key clinical trial trends – EU vs US perspectives

Highlighting the distinct priorities and hurdles faced by sponsors, CROs and sites around the world

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Rare Disease Day 2025 – highlighting recent approvals in the rare disease space

There are more than 7,000 known rare diseases that impact 300 million people globally

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Researchers discover previously unidentified genes linked to rare diseases

Up to 80% of rare disease patients remain undiagnosed after genomic sequencing

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Defying the odds: speeding patient access to life-changing treatments in rare disease

To deliver treatments for rare diseases more quickly, companies share how they are adapting their launch strategies and engaging more deeply with physicians and experts

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Astrategia

Strategy blindness is a thing

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Medscape’s Impact on Women’s Health Education: Key Publications at ISSWSH 2025 & Beyond

Just in time for Women’s History Month and International Women’s Day on March 8th, Medscape is proud to showcase its dedication to advancing women's health education. Sara Thorpe, MPH, Director,...

Medscape Education

Embracing Webcams in Virtual Meetings – Building Company Culture Virtually

Tired of the disconnect in virtual meetings? Discover how using webcams can enhance communication, build stronger relationships, and create a more engaging work environment.

Impetus Digital

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Eli Lilly unveils plans to invest $27bn in four new US manufacturing sites

The investment raises the company’s total US capital expansion commitments to more than $50bn since 2020

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EC approves Celltrion’s RoActemra biosimilar Avtozma for inflammatory diseases

The drug has been approved for rheumatoid arthritis, giant cell arteritis and other indications

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Why local knowledge is key to global pharmacovigilance outcomes

The challenge for companies is to ensure their local affiliates meet local PV obligations while staying aligned with global PV requirements

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