Mark Cohen is a 28 year old lawyer who has a rare eye disease called choroideremia. Its genetic, and his grandpa, brother, and cousin were all diagnosed. He didn't receive…
As reported in Biospace, a new gene therapy has made notable progress towards curing x-linked retinitis pigmentosa, a disease responsible for sight loss in men. Until now, the disease irreversibly…
Tommy Salisbury is eighteen years old. Unlike many teenagers, he is making big moves in the fundraising community and working hard to be a leader in efforts to find…
An article recently published by the Guardian shared the stories of three families affected by recent breakthroughs in gene therapy treatments. What is Gene Therapy? Gene therapy is an increasingly…
According to a scientific study published in the Orphanet Journal of Rare Diseases, a team of researchers were able to identify the mutations responsible for choroideremia in five families of Polish descent.…
4D110 has just received Orphan Drug Designation for the treatment of Choroideremia (CHM). The phase 1 trial of the drug is expected to start in 2019. What is Chorioderemia? Chorioderemia is…
An article by the Evening Standard announces new research towards a cure for cases of genetic blindness. The doctors pursuing this research are able to develop specific parts of the…
According to a story from BioPortfolio, the gene therapy company Nightstar Therapeutics recently released an announcement that the FDA issued Regenerative Medicine Advanced Therapy (RMAT) designation for its investigational product…
Exploding paper-mache volcanoes. Solar systems made of styrofoam balls. Carrot electricity. I remember the sights, smells, and sounds of my high school science fair as it was yesterday. Those earth-shattering…
If you remember reading our story on 12-year-old Markie DeVoe and his choroideremia, we have an important update for you. If you haven't read the story yet, you can find…
What happens when you imagine running a marathon? Unless you are an avid runner, the thought may make you laugh or even cringe. Running even one marathon may seem nearly…
Choroideremia is a disorder that progressively causes you to lose your sight. There is no cure. According to Pinstriped Prospects earlier this year, Markie DeVoe is a 12-year-old living with the…
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