Rare Disease

News

Pfizer takes pipeline blow as it abandons cardiomyopathy dru...

Pfizer has stopped development of a drug to treat cardiomyopathy caused by a rare form of heart disease, after it proved ineffective in a phase 3 trial.

R&D

Study highlights the multibillion dollar burden of rare dise...

Annie Kennedy, chief of policy, advocacy, and patient engagement at the EveryLife Foundation for Rare Diseases, tells us why the Foundation sponsored The National Economic Burden of Rare Di

Views & Analysis

Medical Informatics and Rare Disease: a bridge between two w...

Today’s rare disease landscape comprises of around 7,000 different diseases.

News

Celebrations for ASMD community as EU clears Sanofi's Xenpoz...

Sanofi has claimed approval in the EU for Xenpozyme as a treatment for acid sphingomyelinase deficiency (ASMD) – which becomes the first and only treatment option for patients with the rare

News

West Chester - Circa November 2020: AstraZeneca plant. AstraZeneca has been working on a vaccine for the Coronavirus, COVID and COVID-19.

Data backs AZ's Wilson disease drug, but with a question mar...

Last year, AstraZeneca reported that a new drug to treat Wilson disease developed by its Alexion rare diseases unit had hit the mark in a phase 3 trial, setting it on course for regulatory

News

Sarepta shares yo-yo as FDA places hold on Duchenne trial

Shares in Sarepta came under pressure after the biotech said it had temporarily halted a clinical trial of its new-generation Duchenne muscular dystrophy candidate, after a patient had a se

Pfizer takes pipeline blow as it abandons cardiomyopathy dru...

Study highlights the multibillion dollar burden of rare dise...

Medical Informatics and Rare Disease: a bridge between two w...

Celebrations for ASMD community as EU clears Sanofi's Xenpoz...

Data backs AZ's Wilson disease drug, but with a question mar...

Sarepta shares yo-yo as FDA places hold on Duchenne trial

Bayer gets swift review for Kerendia in type 1 diabetes

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

Rare Disease

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