Parents for the cure of Sickle Cell Disease and Beta-Thalassemia

Parents for the cure of Sickle Cell Disease and Beta-Thalassemia To serve patients and families who suffer from Sickle Cell Disease and Thalassemia by advocating for more affordable treatments.

Dr. Lucio Luzzatto on Gene Therapy for Sickle Cell Disease and Beta Thalassemia
12/01/2021

Dr. Lucio Luzzatto on Gene Therapy for Sickle Cell Disease and Beta Thalassemia

Help start trials for thalassemia and Sickle cell patients for safer and more affordable treatment!
07/23/2021

Help start trials for thalassemia and Sickle cell patients for safer and more affordable treatment!

07/19/2021

Together, Sickle Cell Disease and Beta-Thalassemia represent the most common genetic blood disorders.

Sickle Cell Disease occurs in 1 out of every 365 African American births.

1 out of 12 African Americans carry the trait for Sickle Cell Disease.

The cost per patient for lifetime treatment of Beta-Thalassemia is $4.2 million.

What Does this mean?

With extremely high gene therapy price tags, funding is an enormous obstacle. This leaves patients affected by Sickle Cell Disease and Beta-Thalassemia without access to potential curative therapies.

What if there was an affordable treatment and potential cure available to patients worldwide?

There is…

Researchers at MSK have developed an optimized gene therapy
treatment and potential cure for Sickle Cell Disease and Beta-Thalassemia. This optimized therapy is forecasted to cost $1 million less than competitors. Trials should start as soon as possible.

07/19/2021

Researchers at MSK have developed an optimized gene therapy, showing a promising potential cure for Sickle Cell Disease and Beta-Thalassemia.

With this improved therapy, costs can be reduced by $1 million per patient compared to existing gene therapies.

Researchers at MSK are able to make treatment for those facing Sickle Cell Disease and Beta-Thalassemia more accessible worldwide.

07/17/2021

Researchers at MSK optimized gene therapy is a promising potential cure for Sickle Cell Disease and Beta-Thalassemia
While current treatments, for myeloablation, a harsh conditioning regimen, researchers at MSK will explore a less aggressive conditioning regimen.

Their optimized gene therapy for Sickle Cell Disease and Beta Thalassemia uses the wild-type beta-globin gene and has had no incidences of clonal dominance during the eight years of patient follow-up.

Jonathan Ryan Rashad Wade has Sickle Cell Disease and he voices the importance of why the researchers at MSK, who have developed a promising potential cure for those facing Sickle Cell Disease and Beta-Thalassemia can now have a more effective and affordable treatment.

Help Start clinical trials today!

07/17/2021

What does it mean to be a ? In the first video of our series, warriors from across our community share their stories of advocacy.

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