Understanding The Mechanisms of Charcot-Marie-Tooth Disease Can Help Develop Treatments
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Understanding The Mechanisms of Charcot-Marie-Tooth Disease Can Help Develop Treatments

According to a story from news-medical.net, researchers from The Scripps Research Institute have successfully outlined a new path for the treatment of Charcot-Marie-Tooth disease subtype 2D (CMT2D). The scientists hope…

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A Treatment for Batten Disease Was Given Rare Pediatric Disease Designation from the FDA
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A Treatment for Batten Disease Was Given Rare Pediatric Disease Designation from the FDA

According to a story from pharmpro.com, the pharmaceutical company Abeona Therapeutics announced that it has been given Rare Pediatric Disease Designation for its ABO-202 program by the U.S. Food and…

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The FDA Gave Orphan Drug Designation to an Investigational Treatment For Cystic Fibrosis
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The FDA Gave Orphan Drug Designation to an Investigational Treatment For Cystic Fibrosis

According to a story from Financial Buzz, the pharmaceutical company Proteostasis Therapeutics recently announced that the company's experimental treatment PTI-428 was granted Orphan Drug Designation by the FDA. PTI-428 is…

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Clinical Trials Begin for a Possible Spinocerebellar Ataxia Treatment
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Clinical Trials Begin for a Possible Spinocerebellar Ataxia Treatment

According to a story from pharmavoice.com, the pharmaceutical company Cadent Therapeutics has announced the beginning of the Phase I clinical study for its drug candidate CAD-1883. The therapy is a…

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