2019 Global Genes Rare Patient Advocacy Summit Highlights: Part One

The 2019 Global Genes Rare Patient Advocacy Summit was filled with incredible presentations that highlighted steps for progress in research and advocacy organization, provided solidarity for the rare community, and…

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Eosinophilic Gastrointestinal Disorders Research Receive $7.57 Million In Grant Renewel
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Eosinophilic Gastrointestinal Disorders Research Receive $7.57 Million In Grant Renewel

As originally reported by EurekAlert, eosinophilic gastrointestinal disorder research will receive a boost with the renewal of a $7.57 million grant administered over five years awarded to the Cincinnati Children’s…

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First Patient Dosed in Phase I/II Study of Experimental Gene Therapy for Cystinosis
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First Patient Dosed in Phase I/II Study of Experimental Gene Therapy for Cystinosis

According to a recent press release from the Massachusetts-based biotechnology company Avrobio, Inc., the first patient has been dosed in the Company's phase I/II clinical study of their investigational cystinosis…

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Last Patient Dosed in Phase 2 Study of Experimental ALS Treatment

In a recent press release, Massachusetts-based biotech company Amylyx Pharmaceuticals announced the completion of dosing in the 24 week study of their experimental amyotrophic lateral sclerosis treatment AMX0035. About Amyotrophic…

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