Editor’s Choice: When You Watch This PKU Patient Tell His Story, The Insurance Company Letter Will Make You Furious

Happy Friday, Patient Worthians! This week, we have a video from a PKU patient telling a first-hand account of the deterioration and serious consequences he faced after he went astray…

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A Treatment for Severe Atopic Dermatitis is Considered a Breakthrough by the FDA
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A Treatment for Severe Atopic Dermatitis is Considered a Breakthrough by the FDA

According to a story from biospace.com, a treatment developed by the pharmaceutical company Pfizer was given Breakthrough Therapy designation by the Food and Drug Administration (FDA). The drug is a…

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New Research Is Investigating The Molecular Origins of Primary Ciliary Dyskinesia
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New Research Is Investigating The Molecular Origins of Primary Ciliary Dyskinesia

According to an article from news-medical.net, researchers are beginning to delve into the causes of a rare lung disease called primary ciliary dyskinesia. This research is revealing previously unknown information…

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Gene Therapy Could Be a New Treatment for Muscular Dystrophy
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Gene Therapy Could Be a New Treatment for Muscular Dystrophy

The biotech company Myonexus Therapeutics may soon begin clinical trials for muscular dystrophy gene therapy treatment after obtaining nearly $2.5 million of investments. The research behind the new treatment was…

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Medical-Speak Can Leave Rare Disease Families Feeling Frightened and Intimidated
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Medical-Speak Can Leave Rare Disease Families Feeling Frightened and Intimidated

Nothing prepares families for the myriad of tests with strange sounding names that are required when they start to look for solutions to the problems brought about by a member's…

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Elizabeth Jane Allen, Woman Who Made Lasting Impact on Cystic Fibrosis Community, Dies at 85
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Elizabeth Jane Allen, Woman Who Made Lasting Impact on Cystic Fibrosis Community, Dies at 85

Elizabeth Jane Allen, known best as "Betty", will be honored this week for the wonderful woman she was and the lasting impact she has made on the cystic fibrosis community, reports…

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A New Treatment For Alpha-Mannosidosis Just Got Authorized By The EMA
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A New Treatment For Alpha-Mannosidosis Just Got Authorized By The EMA

According to an announcement from ema.europa.eu, the European Medicines Agency (EMA) recently granted market authorization for a new treatment for alpha-mannosidosis. The new drug is an enzyme replacement therapy called…

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Medicaid Fund Cuts Might Affect Transportation For Those Who Need It
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Medicaid Fund Cuts Might Affect Transportation For Those Who Need It

Maddie Holt from Everett Washington is a 5-year-old with severe disabilities. She can barely hear or see, and she certainly can't walk or talk, reports Maine Public. Unfortunately for her,…

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