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When Parents of Children with Rare Diseases Become Researchers

When Parents of Children with Rare Diseases Become Researchers

When you're told your child has a rare, life-threatening condition you exhaust every resource trying to find answers. But the rarer the condition, the harder those answers are to find…

Continue Reading When Parents of Children with Rare Diseases Become Researchers
Is Gene Therapy The Key for Treating Sickle Cell Anemia?

Is Gene Therapy The Key for Treating Sickle Cell Anemia?

According to a story from Nature, Dr. Elliot Vichinsky thinks that almost a third of his patients with sickle cell disease ultimately die from causes that could be prevented. Sickle…

Continue Reading Is Gene Therapy The Key for Treating Sickle Cell Anemia?
Chinese Researcher’s Gene Editing Project Lands Him in Hot Water, But Could it Lead to Answers for Rare Disease Patients in Future?

Chinese Researcher’s Gene Editing Project Lands Him in Hot Water, But Could it Lead to Answers for Rare Disease Patients in Future?

One Chinese scientist has completed a research project that has shocked many in the medical community. Two twin baby girls born in November of 2018 where the recipients of his…

Continue Reading Chinese Researcher’s Gene Editing Project Lands Him in Hot Water, But Could it Lead to Answers for Rare Disease Patients in Future?
The Patriots Raise Awareness for Sickle Cell Anemia and Duchenne Muscular Dystrophy

The Patriots Raise Awareness for Sickle Cell Anemia and Duchenne Muscular Dystrophy

This past Sunday, December 2nd, during the Patriot's game against the Minnesota Vikings, 20 players wore customized cleats to spread awareness for various philanthropic causes. This is the third year…

Continue Reading The Patriots Raise Awareness for Sickle Cell Anemia and Duchenne Muscular Dystrophy
Finding New Funding Approaches for Rare Disease Drug Development

Finding New Funding Approaches for Rare Disease Drug Development

According to a story from Raconteur, finding sufficient funding for the development of new therapies for rare diseases has always been a major challenge. Rare diseases are defined in the…

Continue Reading Finding New Funding Approaches for Rare Disease Drug Development
Innovative Program in Greenville, SC Helps Patients With Sickle Cell Anemia

Innovative Program in Greenville, SC Helps Patients With Sickle Cell Anemia

According to a story from Greenville Online, the Greenville Health System has begun implementing a comprehensive treatment program for patients with sickle cell disease and its most severe and common…

Continue Reading Innovative Program in Greenville, SC Helps Patients With Sickle Cell Anemia
Is Fetal Gene Therapy the Cure to These Rare Diseases?

Is Fetal Gene Therapy the Cure to These Rare Diseases?

Scientists are researching the possibility of fetal gene therapies as possible cures for rare diseases, such as Duchenne muscular dystrophy, hemophilia, sickle cell disease, and Gaucher's disease.  With fetal gene…

Continue Reading Is Fetal Gene Therapy the Cure to These Rare Diseases?
Resources For Sickle Cell Anemia Patients Are Lacking in Minnesota

Resources For Sickle Cell Anemia Patients Are Lacking in Minnesota

According to a story from MPR News, Rae Baylark, who is the founder and current president of the Sickle Cell Foundation of Minnesota, felt like she had nowhere to seek…

Continue Reading Resources For Sickle Cell Anemia Patients Are Lacking in Minnesota
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*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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