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Editor’s Choice: Approvals and Disappointments in Rare Research News

Editor’s Choice: Approvals and Disappointments in Rare Research News

Happy Thursday Everyone! We hope everyone's spring is off to a good start! This week, we're highlighting four articles about research updates. We have news pertaining to Methylmalonic Acidemia patients…

Continue Reading Editor’s Choice: Approvals and Disappointments in Rare Research News
Enrollment for a Glioblastoma Clinical Trial is Halfway Complete

Enrollment for a Glioblastoma Clinical Trial is Halfway Complete

According to a story from BioSpace, the biopharmaceutical company DelMar Pharmaceuticals, Inc., has recently announced an update to its Phase 2 clinical trial. This clinical trial is testing the experimental…

Continue Reading Enrollment for a Glioblastoma Clinical Trial is Halfway Complete
What is “Stiff Person Syndrome?” A Recent Study Reveals More

What is “Stiff Person Syndrome?” A Recent Study Reveals More

A recent study published in BMC Neurology is revealing more information about an under-diagnosed and poorly understood medical condition called stiff person syndrome. An analysis of several patients that have been…

Continue Reading What is “Stiff Person Syndrome?” A Recent Study Reveals More
Editor’s Choice: Sea Sponges, Reality TV, and Rare Patient Opinions

Editor’s Choice: Sea Sponges, Reality TV, and Rare Patient Opinions

Happy Thursday! We hope everyone's week is going well. Today, we're highlighting a contribution from a cystinosis patient on the necessity of third-party medical providers. We're also sharing a story…

Continue Reading Editor’s Choice: Sea Sponges, Reality TV, and Rare Patient Opinions
National Geographic Features Innovative Research That Will Advance Precision Medicine

National Geographic Features Innovative Research That Will Advance Precision Medicine

Perhaps the most difficult thing about rare disease research is that not only do the conditions affect only a small portion of the population, but like every diagnosis, they affect…

Continue Reading National Geographic Features Innovative Research That Will Advance Precision Medicine
Genetic Screenings in Newborns Can Create Anxious Lives

Genetic Screenings in Newborns Can Create Anxious Lives

A recent article by NBC News is highlighting a growing concern among parents and professionals alike over the effectiveness of newborn genetic screening. There is a certain place between knowing…

Continue Reading Genetic Screenings in Newborns Can Create Anxious Lives
New Research Suggests Many People Aren’t Aware They Carry BRCA Mutations

New Research Suggests Many People Aren’t Aware They Carry BRCA Mutations

A study has found that more than 80% of participants carrying genes linked to breast cancer didn’t know they had them. The research paper, called ‘Exome Sequencing-Based Screening for BRCA1/2…

Continue Reading New Research Suggests Many People Aren’t Aware They Carry BRCA Mutations
An Experimental Blood Test Could Help Determine the Need for Post-Surgical Chemo

An Experimental Blood Test Could Help Determine the Need for Post-Surgical Chemo

According to a story from businesses.com.au, a new blood test that is currently in clinical trials could more accurately determine which patients will need chemotherapy to cure their cancer after…

Continue Reading An Experimental Blood Test Could Help Determine the Need for Post-Surgical Chemo
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Metastatic Breast Cancer: Navigating Grief


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Rethinking What It Means to Live With Acromegaly


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The Let’s Chat CAR T One-on-One Mentor Program: Speaking with Someone Who Understands What You Are Going Through

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