Rare Disease

Views & Analysis

Imparare a convivere con la LHON

Ogni paziente affronta il viaggio della vita a modo proprio, imparando a conoscere la malattia al meglio delle proprie possibilità e attraverso gli strumenti a disposizione.

Views & Analysis

Raising awareness about rare diseases

A family tragedy has prompted Kay Parkinson, founder of the rare disease charity Alstrom Syndrome UK, to create a conference encouraging joined-up thinking to help improve treatments, care and serv

Partner Content

Partner Content

Only 4 Weeks Until Orphan Drugs & Rare Diseases 2018

Pharma Giants Unite to Improve Rare Disease Treatment

Disease Spotlight

Finding the roadmap for living with LHON

Every patient faces their own journey through life, learning about their condition with the best tools available to them.

News

Pfizer axes development of Duchenne muscular dystrophy antib...

Pfizer has axed trials of its domagrozumab antibody for Duchenne muscular dystrophy (DMD), after it failed against efficacy targets.

News

US regulators reject Akcea and Ionis rare disease drug

The US Food and Drug Administration (FDA) rejected Akcea and Ionis’ application for the use of Waylivra in treatment of familial chylomicronemia syndrome (FCS) despite earlier support of ex