Pompe Disease
Efficiency of Pompe Disease Treatment Improved by Molecular “Tag”
By Jodee Redmond from In the Cloud Copy The results of a recent study published in Pompe Disease News suggest that a molecular “tag” used
Rare Patient News. Well Done.
Presentation Highlights Need for Improvements in Familial Hypercholesterolemia Care
- November 21, 2019
FDA Gives Rare Pediatric Disease Designation to Experimental Drug for Treating Rett Syndrome
Study Reveals New Approach for Diagnosing Nonalcoholic Steatohepatitis
A New Treatment for Cushing’s Syndrome Nears EU Approval
LATEST PATIENT WORTHY ARTICLES
Rare Disease
Rare Diseases? They Actually Affect Millions of People
By Rachel Whetstone from In the Cloud Copy Although rare diseases are uncommon by definition, a new study shows that the total number of people
Rare Disease
Why Are Adults in the US Having Trouble Accessing Their Medications?
Modern medication has the ability to save lives and improve living standards. However, medicine is less beneficial when patients’ prescribed treatments are delayed, and entirely
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Myasthenia Gravis
Everything You Want to Know About Myasthenia Gravis
“Myasthenia” means “muscular weakness”. According to a recent article in The Star, studies in Asia indicate that there are approximately five new cases of
Cutaneous T-cell Lymphomas
Company Releases Early Clinical Data For Experimental Blood Cancer Drug
According to a story from pm360online.com, the biopharmaceutical company miRagen Therapeutics, Inc., recently presented Phase I clinical trial data regarding the company’s experimental drug cobomarsen.
Rare Disease
New Research Shows the Importance of Gut Bacteria
Patients in the ICU are under constant surveillance, with consistent monitoring of their blood pressure, blood cultures, and other laboratory markers. These things help doctors
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Why Are Adults in the US Having Trouble Accessing Their Medications?
Modern medication has the ability to save lives and improve living standards. However, medicine is less beneficial when patients’ prescribed treatments are delayed, and entirely
PW Collaborator
November 19, 2019
