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- PMLiVE

CUP

- PMLiVE

Novo Nordisk and Neomorph enter molecular glue degrader partnership worth over $1.4bn

The companies will work on therapies for cardiometabolic and rare diseases

- PMLiVE

Alexion launches ‘colourUp4RARE challenge’ to mark Rare Disease Day 2024

The campaign is aimed at raising awareness of how to improve quality of life for patients

- PMLiVE

Enhancing the patient–HCP relationship in rare diseases

By Maggie Lai

AMICULUM

- PMLiVE

Navigating challenges of MedTech research with rare-disease patients

Authored by: Tom Donnelly, MedTech Director and Amanda Pirraglia, Associate Director. Published in Quirk's Magazine.

Research Partnership

- PMLiVE

Rare disease market access – Launching orphan drugs in secondary markets: the Nordic and MENA regions

Join us live at 1.00pm (GMT)/2.00pm (CET)/8.00am (EST) on Thursday 29th February, to explore rare disease market access challenges and learn more about these high potential markets.

Mtech Access

- PMLiVE

The pitfalls of cutting costs on systematic literature reviews: 11 common issues to avoid

Embarking on a systematic literature review is a critical step in evidence synthesis, providing a foundation for robust decision-making in the healthcare landscape. As with most things, there are ways...

Mtech Access

- PMLiVE

Clinigen’s NaviGATE programme reveals knowledge gaps in rare disease community

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

- PMLiVE

Sanofi expands rare disease pipeline with $2.2bn Inhibrx acquisition

The deal includes a mid-stage candidate for alpha-1 antitrypsin deficiency

Patient Advocate Engagement: A Customer Story

Learn how our client executed a series of 3 virtual touchpoints with 5 rare disease patient advocates to:- Develop a better understanding of the current treatment landscape and lived patient...

Impetus Digital

- PMLiVE

MHRA approves UCB’s Zilbrysq as generalised myasthenia gravis add-on therapy

The autoimmune disease has a global prevalence of 100 to 350 cases per every one million people

- PMLiVE

Takeda’s HyQvia granted FDA approval to treat rare neuromuscular disorder CIDP

Approximately five to seven people per 100,000 in the US are affected by the disorder

On a journey to diverse, inclusive, and more efficient rare disease trials

Although orphan drugs represent a sizeable portion of new drug approvals, most rare diseases still lack effective treatment. Even more so than for other conditions, rare disease management contains large care...

Impetus Digital

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