Mutual Trust Between Doctors and Parents of Children with Rare Diseases Means Optimum Care for the Child

The complexity of rare diseases makes communication between adults involved with the care of these children a vital tool. Communication and mutual trust are of the utmost importance. About the…

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Ipsen Pushes for FDA Approval of Palovarotene for FOP
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Ipsen Pushes for FDA Approval of Palovarotene for FOP

In 2022, the U.S. Food and Drug Administration (FDA) examined the potential of approving palovarotene, a selective RARγ agonist, for the treatment of fibrodysplasia ossificans progressiva (FOP). However, the FDA…

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MDA Clinical & Scientific Conference: Givinostat Improves Physical Function and Mobility in DMD
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MDA Clinical & Scientific Conference: Givinostat Improves Physical Function and Mobility in DMD

The Muscular Dystrophy Association (MDA) recently held its MDA Clinical & Scientific Conference in March 2023. During the conference, stakeholders in the community discussed research trends and clinical practices associated…

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