A Phase 3 Study Shows Encouraging Results for a Potential Treatment for Charcot-Marie-Tooth Type 1A
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A Phase 3 Study Shows Encouraging Results for a Potential Treatment for Charcot-Marie-Tooth Type 1A

Encouraging topline results from a pivotal Phase 3 study into a drug for Charcot-Marie-Tooth type 1A disease have been shared by Pharnext. To find out about this news in more…

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‘Solving Medical Mysteries’: How The Undiagnosed Diseases Network Helps Patients Find Answers
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‘Solving Medical Mysteries’: How The Undiagnosed Diseases Network Helps Patients Find Answers

The Undiagnosed Diseases Network (UDN) is working to help patients with rare undiagnosed conditions find answers. So far, they have helped more than two hundred patients achieve a diagnosis. About…

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Top Misconceptions to Clarify About Achondroplasia (Dwarfism) During Awareness Month

October is Achondroplasia (Dwarfism) Awareness Month! Achondroplasia is a bone growth disorder that causes dwarfism. Dwarfism is defined as a condition of short stature as an adult. People with achondroplasia are…

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This Company Just Endorsed New Patient Advocacy Guidelines
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This Company Just Endorsed New Patient Advocacy Guidelines

According to a story from globenewswire.com, the biopharmaceutical company Orphazyme has officially endorsed a set of guidelines that outline how rare disease patient advocacy organizations and drug companies should interact…

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