Rare diseases

News

Orphazyme 'looking at options' after lead drug hits buffers ...

Orphazyme's hopes of getting EU approval for its Niemann-Pick disease type C (NPC) therapy arimoclomol after a rejection by the US FDA last year are dwindling fast.

Events

Partner Content

Real-World Evidence 2022: Rare Diseases and Innovative Thera...

RWE Live convenes those involved in RWE, HTA and more.

Events

Copy of Bella Mitochondria-Targeted Drug Development - LinkedIn (2)

Partner Content

2nd Mitochondria-Targeted Drug Development Summit

Therapies for mitochondrial dysfunction

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Webinars

The power of R&D collaborations to improve outcomes in rare ...

Far more common than initially thought, rare disease treatments remain far too scarce.

Sales & Marketing

The benefits of being a family-run pharma company

The third generation of his family to hold a leadership role at Chiesi Group, Giacomo Chiesi reflects on the lessons he learned from his father and grandfather - and from his work as a form

Patients

It’s time to do better for patients with rare neurological d...

Global Gene’s Craig Martin introduces a new patient identification initiative that strives to improve diagnosis and access to clinical trials for all patients with rare CNS conditions.