First Patient Dosed in Phase 2 Trial of Experimental Rett Syndrome Treatment
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First Patient Dosed in Phase 2 Trial of Experimental Rett Syndrome Treatment

According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Anavex Life Sciences Corp. has just announced that the first patient has been dosed in its phase 2 clinical trial, which…

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Gene Therapy for 12 Rare Diseases will be Investigated Thanks to this Industry-Academia Partnership

Extended Collaboration  Amicus Therapeutics has just announced that they are expanding their collaboration with the University of Pennsylvania's Perelman School of Medicine for the next five years. This collaboration is…

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Rett Syndrome Trial for a Potential Apnea Treatment will Finish by the End of 2019

Rett Syndrome Rett syndrome is a rare neurodevelopmental disorder. It affects females disproportionately, at a far greater rate than males. It causes cognitive, autonomic, and neurological dysfunction as well as a loss of…

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Company Presents Research Update to Kick off Rett Syndrome Awareness Month
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Company Presents Research Update to Kick off Rett Syndrome Awareness Month

According to a story from Bioportfolio, the biopharmaceutical company Newron Pharmaceuticals has chosen to support Rett Syndrome Awareness Month this year. Rett Syndrome Awareness Months falls on the October of…

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Newron Prepares New Clinical Trial for Rett Syndrome Treatment
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Newron Prepares New Clinical Trial for Rett Syndrome Treatment

Newron Pharmaceuticals S.p.A (Newron) is a biopharmaceutical company specializing in developing inventive therapeutic options. They focus primarily on conditions related to the nervous system. According to bioportfolio.com, Newron announced on…

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Meet the Mom Fundraising for her Two Adult Children with Rare Diseases

I am a caregiver mom to my two special needs, young adult, rare disease kids. I am also a recent widow; previously, I was a caregiver wife to my very…

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Anavex Plans to Trial its Treatment For Parkinson’s Disease Dementia, and Researchers are Hopeful that it Could Also Benefit Other Diseases
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Anavex Plans to Trial its Treatment For Parkinson’s Disease Dementia, and Researchers are Hopeful that it Could Also Benefit Other Diseases

A drug called Anavex 2-73 being developed to treat Parkinson’s disease dementia is planned to undergo testing in a Phase 2 clinical trial, reports Globe Newswire. Parkinson’s disease is a…

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New Research Inches Closer to Treatment For 8-Year-Old Girl And All Rett Syndrome Patients
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New Research Inches Closer to Treatment For 8-Year-Old Girl And All Rett Syndrome Patients

Rett syndrome might limit 8-year-old Laurel Cooper's ability to speak and do every day tasks, but it doesn't hold back her spunky, sassy attitude, reports University of Wisconsin-Madison News. She will…

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AveXis, Gene Therapy Development Company, Expands Program
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AveXis, Gene Therapy Development Company, Expands Program

The gene therapy development company, AveXis, Incorporation, just announced that they will be expanding their clinical trial development program for the potential gene therapy treatment: AVXS-101, reported Globe News Wire.…

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Parents Sue Hospital and Social Services Over Treatment Of Their 11-Year-Old Daughter with Rett Syndrome
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Parents Sue Hospital and Social Services Over Treatment Of Their 11-Year-Old Daughter with Rett Syndrome

The Driscoll family is left devastated and shocked after receiving a letter from social services that states the government could be taking their daughter, who is suffering from Rett syndrome,…

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