A Drug for Pulmonary Arterial Hypertension Will Not be Funded on NHS England
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A Drug for Pulmonary Arterial Hypertension Will Not be Funded on NHS England

According to a story from PharmaTimes, the pulmonary arterial hypertension drug selexipag, developed by Actelion, was unable to make the cut to obtain coverage on the NHS in England. The…

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What’s the Best Way to Meaure Charcot-Marie-Tooth Disease Outcomes?
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What’s the Best Way to Meaure Charcot-Marie-Tooth Disease Outcomes?

Recently in Charcot‐Marie‐Tooth news, progress has been made in identifying better testing to use as outcome measures for people living with Charcot-Marie-Tooth disease type 1A. A study called “Outcome measures…

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Researchers Are Planning to Study the Effects of CBD in Patients with Fragile X
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Researchers Are Planning to Study the Effects of CBD in Patients with Fragile X

Zynerba Pharmaceuticals, Inc. has announced their plans to begin a clinical trial of Cannabidiol in children and teenagers who have Fragile X syndrome. The study, called CONNECT-FX, is expected to…

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MDA Grants $1,000,000 for Charcot-Marie-Tooth Disease Research
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MDA Grants $1,000,000 for Charcot-Marie-Tooth Disease Research

According to a July 9 report by the Muscular Dystrophy Association (MDA), the MDA awarded a large grant to researchers studying Charcot-Marie-Tooth disease. The grant, totaling over $1,000,000, was awarded…

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The European Medicines Agency Has Approved a Paediatric Investigation Plan for an Experimental Treatment of Charcot-Marie-Tooth Disease Type 1A
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The European Medicines Agency Has Approved a Paediatric Investigation Plan for an Experimental Treatment of Charcot-Marie-Tooth Disease Type 1A

The company Pharnext SA, which is developing the investigational drug PXT3003 for the treatment of Charcot-Marie-Tooth disease type 1A, has agreed upon a paediatric investigation plan for the drug with…

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An Eighteen-Minute Documentary Shows How the Frase Family Have Fought for Research into Myotubular Myopathy
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An Eighteen-Minute Documentary Shows How the Frase Family Have Fought for Research into Myotubular Myopathy

The Joshua Frase Foundation has released a new eighteen-minute documentary about the family’s fight for more research into gene therapies for myotubular myopathy, the rare condition that Joshua Frase was…

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