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Rare diseases
Rare diseases
Meet the Expert: Sorena Kiani
Use of ravulizumab for PNH extended to children
Early biologic use in systemic juvenile idiopathic arthritis reduces need for steroids
UK Trust first to provide breakthrough treatment for rare eye condition
Positive CHMP opinion for Akcea orphan drug inotersen
Availability of orphan medicines varies between European countries
Chiesi Group receives European marketing authorisation for Lamzede®
Early access granted for familial chylomicronaemia syndrome drug
Healing Beyond Disease™ initiatives to change people’s lives living with rare diseases
Global initiatives focused on reducing the time to diagnosis of rare diseases announced
Sobi obtains EC approval for new dosing frequency of Orfadin
Abeona Therapeutics Receives Orphan Drug Designation in EU for ABO-102
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Most read
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Transcranial magnetic stimulation brain connectivity changes in major depression visible on MRI scan
by Rod Tucker
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Oral vitamin A could become an alternative to isotretinoin for acne
by Rod Tucker
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Amino acids supplement reduces muscle wasting and improves gut function in critically ill
by Rod Tucker
4
Less than two-thirds of patients alive 10 years after out-of-hospital cardiac arrest
by Rod Tucker
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Launch of the new Hospital Pharmacy Europe Mobile App
by aporter2
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