Recently, gene therapy company Taysha Gene Therapies ("Taysha") announced that its gene therapy candidate, TSHA-102, received both Orphan Drug and Rare Pediatric Disease designations from the FDA. TSHA-102, delivered via…
October is Rett Syndrome Awareness Month, and even though the pandemic has changed the way that we celebrate it, there are plenty of ways to get involved! Rettsyndrome.org has organized…
Female mammals have XX chromosomes and males have XY. But scientists have never understood how females decide which of their two X's should be active in each of its cells.…
What causes developmental disorders? How can the human genome play a role in diagnosing and treating these conditions? Well, a team of scientists from the Salk Institute recently set…
According to a story from Star Tribune, Chelsea Coenraads was 14 months old when her mother Monica became convinced that something seriously wrong with her daughter. She had failed to…
Recently, a team of Yale researchers wanted to understand how genetic mutations in patients with Rett syndrome resulted in neurological issues. Using a unique type of genetic testing, researchers were…
.jpg)
Anavex Life Sciences Corporation has recently announced that they will conduct a clinical trial for their small molecule, ANAVEX®3-71. This therapy is intended to treat neurodegenerative and neurodevelopmental conditions, such…
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…
As originally reported in Angelman Syndrome News, Terry Jo Bichell is a PhD researcher, former director and scientific officer of the Angelman Biomarkers and Outcomes Measures Alliance and Tennessee's ambassador…
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…
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…
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
