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…
Researchers have recently uncovered two new rare diseases caused by the same mutation. This recent discovery is giving more families answers, and should hopefully begin to improve patient outcomes. The…
Rett Syndrome Rett syndrome is a rare and progressive neurodevelopmental disorder caused by mutations to the MECP2 gene. While it affects all ethnic and racial groups, it is found almost…
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…
According to a recent press release by Anavex, the United States Food and Drug Administration has approved an investigational new drug application for a study of the drug Anavex®2-73 in…
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…
If you look on Ed Sheeran's Instagram you will see a recent photo of him and a beautiful eleven-year-old fan. But this is not just any fan-- the picture holds…
According to a story from BioSpace, the biopharmaceutical company AMO Pharma Limited recently announced that the U.S. Food and Drug Administration (FDA) had decided to grant Orphan Drug designation for…
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…
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…
Darwin Ecosystem typically is a name heard in analytic circles. The company specializes in machine-learning and pattern recognition. Now, the company is using their artificial intelligence know-how to help give…
Henry Ford West Bloomfield Hospital is all about their furry friends, AKA their beloved canines, reports Detroit News. If you aren't greeted instantly by Hope and Benson, two of the…
A pre-clinical study of a treatment for Rett syndrome has produced encouraging results, and it is hoped that this will lead to further research. The full story can be read…
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…
CNN published this incredibly story about love and bravery earlier this month - and it's worth a second look. Sophia Weaver was born with deformities to her hands, feet, and…
NBCNews Chief Foreign Correspondent, Richard Engel, opened up about one of the hardest phone calls he ever had to receive-- the call where he received the diagnosis of Rett syndrome for…
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…
According to a story from PR Newswire, the pharmaceutical company Biohaven announced that they have received clearance from the Food and Drug Administration (FDA) to begin work on its clinical trials…
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.…
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,…
Rett syndrome is a severe, genetic neurological disorder, which primarily affects girls. While it was once thought to be a form of autism, it's now understood as its own condition.…
Recent research has found new discoveries for those with neurodevelopmental disorders like Rett Syndrome. These new findings offer hope that those with mental disabilities like autism do not necessarily have…
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