Study: ANAVEX 3-71 as a Treatment for Neurodegenerative Diseases
source: pixabay.com

Study: ANAVEX 3-71 as a Treatment for Neurodegenerative Diseases

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…

Continue Reading Study: ANAVEX 3-71 as a Treatment for Neurodegenerative Diseases
After Civil War Drove Her From Her Homeland, She Went on to Discover the Genes Causing Spinocerebellar Ataxia and Rett Syndrome
source: pixabay.com

After Civil War Drove Her From Her Homeland, She Went on to Discover the Genes Causing Spinocerebellar Ataxia and Rett Syndrome

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…

Continue Reading After Civil War Drove Her From Her Homeland, She Went on to Discover the Genes Causing Spinocerebellar Ataxia and Rett Syndrome
Investigational Treatment for Rett Syndrome gets Rare Pediatric Disease Designation
source: pixabay.com

Investigational Treatment for Rett Syndrome gets Rare Pediatric Disease Designation

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…

Continue Reading Investigational Treatment for Rett Syndrome gets Rare Pediatric Disease Designation

Trofinetide Granted Rare Pediatric Disease Designation for the Treatment of Rett Syndrome

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…

Continue Reading Trofinetide Granted Rare Pediatric Disease Designation for the Treatment of Rett Syndrome
These Two Companies Just Announced a Partnership to Develop Multiple Gene Therapies
EliasSch / Pixabay

These Two Companies Just Announced a Partnership to Develop Multiple Gene Therapies

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…

Continue Reading These Two Companies Just Announced a Partnership to Develop Multiple Gene Therapies
An Experimental Treatment for Rett Syndrome Earns Rare Pediatric Disease Designation
source: pixabay.com

An Experimental Treatment for Rett Syndrome Earns Rare Pediatric Disease Designation

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…

Continue Reading An Experimental Treatment for Rett Syndrome Earns Rare Pediatric Disease Designation
First Ever International Burden of Illness Survey for Rett Syndrome to be Conducted
TeroVesalainen / Pixabay

First Ever International Burden of Illness Survey for Rett Syndrome to be Conducted

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…

Continue Reading First Ever International Burden of Illness Survey for Rett Syndrome to be Conducted

Chief Foreign War Correspondent at NBC News Gives Account of His 4-Year-Old Son With Rett Syndrome

  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…

Continue Reading Chief Foreign War Correspondent at NBC News Gives Account of His 4-Year-Old Son With Rett Syndrome
Independent Data Review Gives Go-Ahead for Rett Syndrome Clinical Trial
jarmoluk / Pixabay

Independent Data Review Gives Go-Ahead for Rett Syndrome Clinical Trial

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…

Continue Reading Independent Data Review Gives Go-Ahead for Rett Syndrome Clinical Trial
First Patient Dosed in Phase 2 Trial of Experimental Rett Syndrome Treatment
mcmurryjulie / Pixabay

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…

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

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…

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

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…

Continue Reading Rett Syndrome Trial for a Potential Apnea Treatment will Finish by the End of 2019
Company Presents Research Update to Kick off Rett Syndrome Awareness Month
Cozendo / Pixabay

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…

Continue Reading Company Presents Research Update to Kick off Rett Syndrome Awareness Month
Close Menu