Healing Genes

06/19/2021

Sickle Cell awareness day June 19, 2021.
Why some are hopeful about CRISPR technology for SCD

The success of a Mississippi woman's treatment for sickle cell disease provides hope that gene editing could help treat the ailment

04/01/2021

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.

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....

03/01/2021

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

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...

02/24/2021

Justina Williams, living with sickle cell disease and working in support of cures, reminded me today of the importance of giving blood, even in COVID-19 times. If you can, please give. I gave with the American Red Cross who make it easy, use good safety protocols, and remind you so you don't miss your appointment. It's critical!

01/04/2021

If I Have a Rare Disease, Will the Vaccine Affect My Eligibility to Receive Gene Therapy in the Future?
If you are immunocompromised, speak with your provider to make the decision about getting vaccinated. However, there is no use of a virus or viral vector with the mRNA vaccines, so receiving an mRNA vaccine will not prevent you from obtaining a gene therapy in the future for your rare disease.

For more FAQ on the COVID-19 vaccines

The use of messenger RNA (mRNA) in medical interventions has been researched for the last 20 years, including in vaccines hoping to fight diseases such as cancer, rabies, Ebola and the Zika virus. That means the research was ready to be applied to this new disease, COVID-19. With the recent authoriz...

12/02/2020

RARE-X is going to demonstrate how their new approach to patient-owned data collection and federated data sharing for biomedical research will accelerate disease understanding and therapy development. they just got the funding they need to start their 24 month pilot period.

Good data changes lives.

First global patient-powered data collection, analysis, and sharing platform will accelerate rare disease research and collaboration Aliso Viejo, CA — Dec 2, 2020 — RARE-X, a collaborative platform for patient-controlled and structured data collection, global data sharing and analysis that will ...

12/02/2020

Our friends at Rare-X RARE-X are building the largest patient-driven, collaborative, open-data access project for rare diseases globally. visit

We believe that rare patient communities, equipped to securely gather and share their data with collaborating researchers and companies around the globe, will significantly expand diversity and inclusion in research and dramatically accelerate progress across rare diseases.

10/26/2020

there is a nice news story about baby Lucy in Canada who has SMA - Spinal Muscular Atrophy. She has been able to receive Zolgensma, a gene therapy that is approved in the US but not in Canada, where Lucy lives.

The treatment is very expensive. The parents were using GoFundMe to save money to pay for it.

Since Lucy will now receive the Gene Medicine for free, some of the funds raised can go to other families with SMA. We wish the best for baby Lucy, her brother, and her parents.

A Canadian family that spent months frantically raising millions of dollars for a one-time gene therapy treatment to save their daughter's life has received the treatment for free.

10/14/2020

Here's a story of resilience from Jake Javier who hit his head in a pool and broke his neck. He was a recipient of a special experimental treatment in a clinical trial using stem cells, and he regained function in his arms and legs.

An accident left Jake Javier paralyzed from the chest down the day before his high school graduation. Through a clinical trial, Javier received a transplant of 10 million stem cells and regained use of his arms and hands. Javier shares his story of resilience, science, and hope.