Healing Genes

Sickle Cell awareness day June 19, 2021.
Why some are hopeful about CRISPR technology for SCD https://www.healthline.com/health-news/first-person-treated-for-sickle-cell-disease-with-crispr-is-doing-well

First Person Treated for Sickle Cell Disease with CRISPR Doing Well The success of a Mississippi woman's treatment for sickle cell disease provides hope that gene editing could help treat the ailment

Scholarship Fund offers awards of $5,000 each for Fall 2021. Deadline is May 7, 2021 - for any rare disease patient pursuing accredited classes at any stage in their life. https://everylifefoundation.org/rare-scholarship/?eType=EmailBlastContent&eId=b4d4c80f-0e8a-46cd-a946-d4e412db2d7c

Rare Scholarship | EveryLife Foundation for Rare Diseases Living with a rare disease means managing unique challenges, including frequent doctor visits, rigorous treatment regimens and hospitalizations, and exposure risks. While quality and duration of life continues to improve thanks to improved diagnosis and treatment approaches, individuals living with....

How does your patient experience help bring about cell- and gene- medicine therapies? When it comes to personal patient info about rare diseases,
how do you collect data, structure it, adhere to rigorous standards, and share it responsibly?

Some answers are found in a new paper from Rare-X created in association with the ARM Foundation for Cell and Gene Medicine.
https://rare-x.org/case-studies/enabling-the-future-of-cell-gene-therapies-through-non-proprietary-patient-owned-data-collection/

read “Enabling the Future of Cell & Gene Therapies through Non-Proprietary Patient-Owned Data Collection” from Rare-X, Feb 2021

Enabling the Future of Cell & Gene Therapies through Non-Proprietary Patient-Owned Data Collection - RARE-X White Paper Enabling the Future of Cell & Gene Therapies through Non-Proprietary Patient-Owned Data Collection This paper explores the primary issues and requirements for data gathering that cell and gene therapy developers face. It also considers solutions, and the role a standardized non-proprieta...