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A Teenager was Killed by a Disease so Rare it Didn’t Have a Name, But His Story Could Change the Lives of Others Like Him

A Teenager was Killed by a Disease so Rare it Didn’t Have a Name, But His Story Could Change the Lives of Others Like Him

According to a story from NBC News, Mitchell Herndon, a 19 year old from Missouri, recently passed away due to the progression of a rare genetic disease that is so…

Continue Reading A Teenager was Killed by a Disease so Rare it Didn’t Have a Name, But His Story Could Change the Lives of Others Like Him
Enrollment Begins for Amyotrophic Lateral Sclerosis Clinical Trial

Enrollment Begins for Amyotrophic Lateral Sclerosis Clinical Trial

According to a story from BioPortfolio, the biopharmaceutical company Collaborative Medicinal Development, LLC has recently announced that the first patient has been enrolled in a clinical trial that will test…

Continue Reading Enrollment Begins for Amyotrophic Lateral Sclerosis Clinical Trial
Editor’s Choice: Rare Patient Innovation and Support

Editor’s Choice: Rare Patient Innovation and Support

Happy Thursday everyone! This week, we're highlights two stories about patients taking action to solve problems in their life: one patient innovates to improve their care, and another starts a…

Continue Reading Editor’s Choice: Rare Patient Innovation and Support
A Primer on Clinical Trials for Patients With Gaucher Disease and Other Rare Illnesses

A Primer on Clinical Trials for Patients With Gaucher Disease and Other Rare Illnesses

According to a post from gaucherdisease.org, clinical trials are an essential component to the development and testing of new therapies for Gaucher disease and other rare diseases. For some rare…

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Gene Editing Stem Cells Eliminates Their Chance of Rejection, Study Finds

Gene Editing Stem Cells Eliminates Their Chance of Rejection, Study Finds

A recent study, published in Nature, examined whether or not stem cells could be modified to reduce their chance of rejection. Stem cell therapies are utilized in a wide array…

Continue Reading Gene Editing Stem Cells Eliminates Their Chance of Rejection, Study Finds
Independent Data Review Gives Go-Ahead for Rett Syndrome Clinical Trial

Independent Data Review Gives Go-Ahead for Rett Syndrome Clinical Trial

According to a story from Financial Buzz, the biopharmaceutical company Anavex Life Sciences Corp. has recently announced that the Independent Data Safety Monitoring Board (DSMB) has completed its review of…

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NeuroGen to Host No-Charge Outpatient Stem Cell Therapy Consultation Camp in Chandigarh

NeuroGen to Host No-Charge Outpatient Stem Cell Therapy Consultation Camp in Chandigarh

According to a publication from NewZNew, the NeuroGen Brain and Spine Institute in India plans on hosting an outpatient stem cell therapy consultation camp in Chandigarh on the last day…

Continue Reading NeuroGen to Host No-Charge Outpatient Stem Cell Therapy Consultation Camp in Chandigarh
Tanzanian Girl With Gaucher Disease to Receive Treatment Through Critical Program

Tanzanian Girl With Gaucher Disease to Receive Treatment Through Critical Program

According to a story from Direct Relief, when Yusra was just a year old, she began to have mysterious bouts of fever. Her family had no explanation. By age three,…

Continue Reading Tanzanian Girl With Gaucher Disease to Receive Treatment Through Critical Program
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The Mentor She Wished She Had - How Elizabeth Became a Lifeline for EB
Finding Strength Together: Scott and Katie’s Journey with Advanced Kidney
You Are Not Alone: Empowering the Advanced Kidney Cancer Community
Finding Light Through Story-The Power of Ambassadorship in the Endometrial Cancer Community
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