Grant to Fund Critical International Amyotrophic Lateral Sclerosis Research
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Grant to Fund Critical International Amyotrophic Lateral Sclerosis Research

According to a story from med.miami.edu, an international research consortium known as Clinical Research in ALS and Related Disorders for Therapeutic Development (CReATe) has just been awarded a renewal grant from…

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Personalized Medicine: A Drug Tailored for a Six Year Old With Batten Disease May Serve as a Template for Similar Rare Disorders

  Mila Makovec began her life as a normal, healthy infant, and according to a recent article in Science Magazine, she continued along this path until reaching the age of…

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The FDA Has Just Approved the Very First Therapy for Erythropoietic Protoporphyria

EPP Erythropoietic protoporphyria (EPP) is a rare disease that causes extreme light sensitivity. This sensitivity is so extreme that being outside is extremely difficult for patients and many only come…

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Experimental Facioscapulohumeral Dystrophy Drug Found Safe and Tolerable in Phase 1 Study
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Experimental Facioscapulohumeral Dystrophy Drug Found Safe and Tolerable in Phase 1 Study

A recent press release from Fulcrum Therapeutics, a Massachusetts-based biotechnology company, announced preliminary findings of the company's phase 1 clinical study of losmapimod in the treatment of facioscapulohumeral dystrophy. Losmapimod is…

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An Experimental Cystic Fibrosis Treatment Could Make This Patient’s Greatest Wish Come True

According to a story from abc.net.au, Darren Bullock is getting pretty long in the tooth---at least for a cystic fibrosis patient. He is 44 years old, and his lungs currently…

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