Rare Disease

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Boehringer Ingelheim, Biberach, Germany, February 2007

Boehringer bags first approval for rare skin disease drug

German drugmaker Boehringer Ingelheim has its first regulatory approval for spesolimab, its antibody therapy for rare and life-threatening skin disorder generalised pustular psoriasis (GPP)

News

Xenpozyme is first FDA-approved drug for rare disease ASMD

Sanofi now has approval on both sides of the Atlantic for Xenpozyme – its enzyme replacement therapy for rare disease acid sphingomyelinase deficiency (ASMD) – after getting a green light f

News

CSL preps filings for once-monthly antibody for HAE

Australia's CSL has said its one-monthly treatment for hereditary angioedema has shown it is safe and effective in a phase 3 trial, and will be filed for approval in its current financial y

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CSL Vifor plans UK launch after NICE backs rare disease drug...

UK cost-effectiveness organisation NICE has recommended routine NHS use of a recently-approved therapy for two forms of ANCA-associated vasculitis (AAV) – oral complement C5 inhibitor Tavne

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

Boehringer bags first approval for rare skin disease drug

Xenpozyme is first FDA-approved drug for rare disease ASMD

CSL preps filings for once-monthly antibody for HAE

CSL Vifor plans UK launch after NICE backs rare disease drug...

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

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

Consolidating data strategy for global success: Insights fro...

How to meet your physicians where they are online

Rare Disease

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