As originally reported in Discover Magazine, Huda Zoghbi took an unexpected route to winning the Breakthrough Prize in Life Sciences in 2017 for her discoveries of the genes responsible for…
According to a story from Wapakoneta Daily News, the drug companies ACADIA Pharmaceuticals Inc. and Neuren Pharmaceuticals Limited have recently announced that its investigational product candidate trofinetide has earned Rare…
.jpg)
ACADIA Pharmaceuticals, working with Neuren Pharmaceuticals, has recently been granted the Rare Pediatric Disease designation from the FDA for their therapy Trofinetide. This drug is intended for the treatment of…
As originally reported in Financial Buzz, Rett Syndrome has received promising news: a drug undergoing review, ANAVEX®2-73 (blarcamesine), has received fast track designation, meaning patients may be able to receive…
Copyscape score: 4% A common symptom of Rett Syndrome is apnea, which is a difficulty in breathing. This apnea has been a symptom that has had no treatment for a…
According to a story from BioSpace, the gene therapy companies Sarepta Therapeutics, Inc. and StrideBio, Inc. have recently announced the completion of a licensing and collaboration agreement that could lead…
According to a story from marketscreener.com, the biopharmaceutical company Anavex Life Sciences Corp. has announced that its experimental product candidate ANAVEX2-73 has earned Rare Pediatric Disease designation from the US…
According to a story from BioSpace, the biopharmaceutical company Newron Pharmaceuticals S.p.A. has announced that is partnering with the Rett syndrome disease community in order to sponsor an all new…
As originally reported in SF Gate, Jeannette and Victor Vega quickly realized that taking care of their second daughter, Tiana, was not going to be so easy this time. From…
According to a story from Belfast Live, parents Tara and Glenn Blaine didn't take long to recognize that their newborn daughter Mia was not developing normally. Ultimately, the little girl…
According to a story from Spectrum News a recent study revealed two potential therapies that could treat symptoms associated with Rett syndrome, a rare disorder. The study involved the creation…
.jpg)
Richard Engel’s son Henry, age 4, has been diagnosed with Rett syndrome, a thus far incurable genetic neurological disease. Engel is chief foreign war correspondent at NBC and has…
According to a story from Financial Buzz, the biopharmaceutical company Anavex Life Sciences Corp. has recently announced that the Independent Data Safety Monitoring Board (DSMB) has completed its review of…
According to a story from EurekAlert! an international team of scientific researchers have made an unusual discovery. The team found in the course of their studies that dietary supplementation with…
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…
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…
© Copyright Patient Worthy
