Healing Genes

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.

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....

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!

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

mRNA Vaccines for COVID-19 | 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...

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.

RARE-X, Launches First Research Pilot Projects For Rare Disease 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 ...

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 family that raised $2 million for their baby's life-saving medical treatment has received it for free 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.