Supporting the LCA and rare retinal disease community
We see a cure for blindness.
What is Leber Congenital Amaurosis (LCA)?
LCA is a rare inherited retinal disease characterized by severe vision loss at birth. While some children are born with little or no vision, others may have significant vision loss in the first few years of life, stable vision for a period of time, and then eventually complete vision loss as the retina deteriorates into total blindness.
What We Do
Founded in 2014, Sofia Sees Hope is a nonprofit 501(c)3 patient advocacy organization dedicated to generating awareness, raising funds for research, and providing support, education and outreach to the LCA and rare inherited retinal disease community.
Thank You to These Organizations
for their Ongoing Commitment to Sofia Sees Hope.
Let’s Get Social!
Our "Your Voice Matters” panel at our 7/27 LCA Family Conference brings together patient advocates from @spark_tx @AGTC_ @BarthSyndrome and the #sicklecell community. Register: https://t.co/tpqj4ojTMS…/sofiaseeshope2019/925219/ #LCA #IRD
Pioneering LCA patient Tami Morehouse at LCA Family Conference July 27 in Philly for a panel called "All About Clinical Trials” w/ Dr. Wiley Chambers of @US_FDA & Michel Michaelides of @MeiraGTX. https://t.co/lrvsG20yGM
. @fightblindness recently published in @Genes_MDPI on its role in driving genetic research for Inherited Retinal Diseases. Read: https://t.co/Wg4gUmp1f2. We are hosting FFB CEO Ben Yerxa at our LCA Family Conference in Philadelphia, 7/27.Info: https://t.co/lrvsG20yGM
If you are hunting for bargains online today - please choose to support Sofia Sees Hope! Thank you! https://t.co/n6swdBIwod
One phrase you will hear us say over and over (and ditto our friends at National Organization for Rare Disorders, Inc. (NORD) - is Your Voice Matters! Patient advocacy is *so critical* to making sure important legislation is enacted, and that research and clinical trials move forward.
We are excited to present these panelists for "Your Voice Matters" at our July 27 LCA Family Conference in Philadelphia:
~ Jamie Ring, Head, Patient Advocacy at Spark Therapeutics.
~ Jill Dolgin, Head of Patient Advocacy at AGTC
~ Emily Milligan, Executive Director at the Barth Syndrome Foundation
~ Terri Booker, Advocate for Sickle Cell Anemia
Moderated by Annette Tonti, Executive Director of Sofia Sees Hope
Register for the conference today: www.eiseverywhere.com/ehome/sofiaseeshope2019/925219/
Dr. Katherine A. High is co-founder, president, and chief scientific officer of Spark Therapeutics. We are so pleased to welcome her to our LCA Family Conference in Philadelphia on Saturday, July 27. Her talk is entitled "The Long and Winding Road of the Human Genome Project and Gene Therapy."
In a recent interview, Dr. High said, "There were many disappointments on the nearly 20-year road to developing that gene therapy for hemophilia and publishing those exciting results. In each case, I tried to carefully analyze the situation so that I could learn what went wrong and then move forward – and try to avoid repeating those mistakes. When something that you believe in and have personal conviction about doesn’t work out, it’s hard, but you have to be persistent."
Learn more about our conference (and register), which also includes representatives from pharma, industry, researchers, regulators and patients. www.eiseverywhere.com/ehome/sofiaseeshope2019/925219/
www.ashclinicalnews.org/features/pulling-back-curtain-katherine-high-md/
Pulling Back the Curtain: Katherine A. High, MD - ASH Clinical News
Pulling Back the Curtain: Katherine A. High, MD - Features, Pulling Back the Curtain - ASH Clinical News