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The NFL’s “My Cause My Cleats”: Which Teams Raised Rare Disease Awareness?

The NFL’s “My Cause My Cleats”: Which Teams Raised Rare Disease Awareness?

Week 13 of the NFL marks the onset of “My Cause My Cleats,” an annual initiative that empowers players to reveal and share their passions beyond the game. This year…

Continue Reading The NFL’s “My Cause My Cleats”: Which Teams Raised Rare Disease Awareness?
Evaluating ST-920 for Fabry Disease: An Interview with Sangamo’s Bettina Cockroft, MD, MBA

Evaluating ST-920 for Fabry Disease: An Interview with Sangamo’s Bettina Cockroft, MD, MBA

The European Society for Gene and Cell Therapy (ESGCT) recently held its Annual Congress from October 11-14, 2022. During the ESGCT Congress, various stakeholders discussed data, trends, and new research…

Continue Reading Evaluating ST-920 for Fabry Disease: An Interview with Sangamo’s Bettina Cockroft, MD, MBA
Editor’s Choice: A New Dystonia Center of Excellence, A Call for Sickle Cell Blood Donors, and a New Treatment Target for Glioblastoma

Editor’s Choice: A New Dystonia Center of Excellence, A Call for Sickle Cell Blood Donors, and a New Treatment Target for Glioblastoma

Happy Friday! A tropical storm system will be traveling up through the mid-Atlantic this weekend, bringing wind and rain. Hunker down! This week, we have stories on Duke University being…

Continue Reading Editor’s Choice: A New Dystonia Center of Excellence, A Call for Sickle Cell Blood Donors, and a New Treatment Target for Glioblastoma

Dreamsickle Kids Foundation’s 5th Annual Las Vegas Sickle Cell Walk

The 5th Annual Las Vegas Sickle Cell Walk September 24, 2022 Dreamsickle Kids Foundation 5th Annual Las Vegas Sickle Cell Walk, this event is the only fundraiser we have to…

Continue Reading Dreamsickle Kids Foundation’s 5th Annual Las Vegas Sickle Cell Walk
Experimental Stem Cell Treatment Benefits Girl with PAH

Experimental Stem Cell Treatment Benefits Girl with PAH

  Pulmonary arterial hypertension (PAH) is a chronic and progressive form of high blood pressure in the lungs which can result from a number of causes: HIV, sickle cell disease,…

Continue Reading Experimental Stem Cell Treatment Benefits Girl with PAH
FDA Grants Rare Pediatric Disease Designation for Experimental Beta Thalassemia Drug

FDA Grants Rare Pediatric Disease Designation for Experimental Beta Thalassemia Drug

EDIT-301 is an investigational, gene editing medicine developed by Editas Medicine for the treatment of beta thalassemia. As recently reported in the Business Insider, EDIT-301 has just been granted the…

Continue Reading FDA Grants Rare Pediatric Disease Designation for Experimental Beta Thalassemia Drug
EDIT-301 for Beta Thalassemia Earns Rare Pediatric Disease Designation

EDIT-301 for Beta Thalassemia Earns Rare Pediatric Disease Designation

Rare Pediatric Disease designation is granted by the FDA to drugs or biologics intended to treat life-threatening diseases affecting individuals aged 18 or younger. In this case, rare conditions are…

Continue Reading EDIT-301 for Beta Thalassemia Earns Rare Pediatric Disease Designation
Rare Disease Week 2022: How to Become the Hero in Someone Else’s Story

Rare Disease Week 2022: How to Become the Hero in Someone Else’s Story

Rare Disease Week 2022 Rare Disease Week is the week that leads up to Rare Disease Day, which is recognized on the last day of February. This year, we are…

Continue Reading Rare Disease Week 2022: How to Become the Hero in Someone Else’s Story
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Finding Light Through Story-The Power of Ambassadorship in the Endometrial Cancer Community
*cancer that cannot be removed with surgery or has spread to other parts of the body. GEJ= gastroesophageal junction (where the esophagus joins the stomach)
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