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The New Gold Rush in Rare Disease Treatments and Merck KGaA’s Acquisition of SpringWorks Therapeutics
For many years, the pharmaceutical business concentrated mostly on blockbuster medications, which are cures for common ...
‘That could be your family member’: Rare diseases affect 30 million Americans
The National Organization for Rare Disorders is a coalition of patient advocacy groups and the first national nonprofit that ...
pharmaphorum5h
Paediatric rare diseases: Children are not small adults
Around 75% of rare diseases are diagnosed in childhood, with most before the age of two. With 30% of rare disease patients ...
News Medical on MSN2d
Rare enterovirus strain in Spain raises alarms over potential outbreaks
Scientists in Spain detected Enterovirus C105 (EV-C105), a rare virus linked to respiratory and possible neurological illness ...
Scientists Describe Rare Syndrome Following Covid Vaccinations
In a small study, patients with the syndrome were more likely to experience reactivation of Epstein-Barr virus and high ...
Innovations in research and treatment for rare diseases
More than 30 million people in the United States are affected by rare diseases, many of which are life-threatening conditions ...
Acsh.org9d
Rare Diseases Are Neglected. The FDA Needs A Dedicated 'Center of Excellence for Rare Diseases'.
The FDA has made some modest efforts, but it needs an internal organization that understands and would be an aggressive ...
Channel NewsAsia Singapore3h
Daily Cuts - Carrying Hope for those living with rare diseases
For patients and caregivers living with rare diseases, life can be a daily struggle. Ahead of World Rare Disease Day this ...
Johns Hopkins Medicine3d
For Patients with Rare Diseases, Genetic Counselors Can Save Lives
As key members of the Johns Hopkins Department of Genetic Medicine, genetic counselors help patients with rare diseases ...