Fragile X Syndrome: Innovative Approaches to Finding a Cure

On World Fragile X Day, July 22, FRAXA co-hosted the special virtual event, “Fragile X Syndrome: Innovative Approaches to Finding a Cure,” with WuXi AppTec. Together we provided a global stage where leading researchers, drug developers and physicians shared their latest discoveries aimed at treating and ultimately curing this syndrome.

Special thanks to our 30+ distinguished speakers, and our supportive partners: Fragile X Association of Australia, Fragile X Association of China, Fragile X Society India, Fragile X Syndrome Parent’s Group Argentina, Fragile X South Africa, and Fragile X Center Georgia.

---- Chapters ----
0:00:00 - Intro
0:02:13 - Hui Cai (WuXi Apptec) – Welcome
0:04:37 - Katie Clapp (FRAXA) – Welcome
0:06:36 - Elizabeth Berry-Kravis (RUSH University Med Ctr), Peter Kind (University of Edinburgh), Michael Tranfaglia (FRAXA) – Opening Plenary
0:32:14 - Wendy Bruce (FX Assoc. of ASTL), Jeannie Lee (Harvard) – Gene Reactivation
0:42:04 - Kathryn Whitehead (Carnegie Mellon Univ.) – mRNA Replacement
0:44:36 - David Hampson (Univ of Toronto) – Gene Therapy
0:46:07 - Ralph Laufer (Lysogene) – Gene Therapy
0:47:40 - Ernest Pedapati (Univ. of Cincinnati) – Protein Replacement
0:49:35 - Mark Bear (MIT), Craig Erickson (Univ. of Cincinnati) – Targeted Therapy
1:03:38 - Stephen O’Quinn (Zynerba) – Targeted Therapy
1:05:10 - Adrian Newman-Tencredi (Neurolixis) – Targeted Therapy
1:06:54 - Lili Mao (GexVal) – Targeted Therapy
1:08:59 - Daniel Alkon (Synaptogenix) – Targeted Therapy
1:10:18 - John Atack (Cardiff University) – Targeted Therapy
1:12:11 - Mara Dierssen (Centre for Genomic Regulation) – Targeted Therapy
1:14:49 - Lynn Durham (Stalicla) – Targeted Therapy
1:17:01 - Christopher Missling (Anavex) – Targeted Therapy
1:18:44 - Robert Ring (Kaerus) – Targeted Therapy
1:20:32 - Fabio Tucci (Epigen Biosciences) – Targeted Therapy
1:22:10 - David Walker (Sentinel Oncology) – Targeted Therapy
1:23:14 - Bruce Bloom (Healx), Andres Centellas (FRAXA) – Drug Repurposing
1:35:00 - Michael Hayden (Prilenia) – Drug Repurposing
1:37:54 - Mahmoud Pouladi (Univ. of British Columbia) – Drug Repurposing
1:38:43 - Randall Carpenter (Allos Pharma) – Drug Repurposing
1:40:20 - Isaac Bentwich (Quris-AI) – Drug Repurposing
1:42:07 - George Drakakis (Purposeful) – Drug Repurposing
1:43:30 - Nik Subramanian (Kantify) – Drug Repurposing
1:45:28 - Gordon Wang (Stanford Univ) – Drug Repurposing
1:47:27 - Xinyu Zhao (Univ of Wisconsin) – Drug Repurposing
1:48:20 - Richard Soll (WuXi Apptec) – Closing Remarks

FRAXA’s mission is to find effective treatments and ultimately a cure for Fragile X syndrome. We directly fund research grants and fellowships at top universities around the world. We partner with biomedical and pharmaceutical companies, large and small, to bridge the gap between research discoveries and actual treatments.

Treatments for Fragile X are likely to help people affected by autism, Alzheimer’s, and other brain disorders.

To learn more about FRAXA's Fragile X syndrome research, to get involved with Fragile X awareness and events or to donate to FRAXA research, visit www.fraxa.org

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