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This New Discovery About Magnesium Could Have Implications for Rare Disease Treatment

This New Discovery About Magnesium Could Have Implications for Rare Disease Treatment

According to a story from UT Health San Antonio, a team of researchers have discovered in a new study the mechanism behind the activation of magnesium ions in cells. The…

Continue Reading This New Discovery About Magnesium Could Have Implications for Rare Disease Treatment

Can We Be Cautiously Optimistic About Regenerating Damaged Optic Nerve Cells in Mice?

According to a recent article in ScienceAlert, scientists have conducted experiments in mice activating a protein called protrudin that gives damaged nerve fibers the ability to regenerate. Currently, once nerve…

Continue Reading Can We Be Cautiously Optimistic About Regenerating Damaged Optic Nerve Cells in Mice?
TLMdX Accepts Late-Breaking Results on Odevixibat for PFIC

TLMdX Accepts Late-Breaking Results on Odevixibat for PFIC

Later this week, the American Association for the Study of Liver Diseases (AASLD) will hold its virtual The Liver Meeting Digital Experience (TLMdX). Altogether, TLMdX will last from November 13-16.…

Continue Reading TLMdX Accepts Late-Breaking Results on Odevixibat for PFIC
Experimental Treatment for Acid Sphingomyelinase Deficiency Shows Potential in Trials

Experimental Treatment for Acid Sphingomyelinase Deficiency Shows Potential in Trials

In a press release from Sanofi, a global biopharmaceutical company, the company's experimental therapy candidate olipudase alfa has demonstrated the ability to substantially improve spleen volume and lung function in…

Continue Reading Experimental Treatment for Acid Sphingomyelinase Deficiency Shows Potential in Trials
COVID-19 Vaccine Trial Resumes in the US

COVID-19 Vaccine Trial Resumes in the US

Currently, there are a number of vaccines for COVID-19 that are being investigated, one of which is AZD1222. The trial was paused back in September when a participant became ill…

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Ataluren Halts Disease Progression in Patients with nmDMD

Ataluren Halts Disease Progression in Patients with nmDMD

This year, at the 2020 CNS-ICNA Conjoint Meeting, researchers identified ataluren as a potential treatment that inhibits disease progression for patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Research suggests…

Continue Reading Ataluren Halts Disease Progression in Patients with nmDMD
Positive Results of Phase 2 Trial for Atopic Dermatitis Announced

Positive Results of Phase 2 Trial for Atopic Dermatitis Announced

Positive results have just been announced for a Phase 2 study called QUARTZ2, investigating SHR0302 as a therapy for atopic dermatitis (AD). Researchers found  that at every dose level examined,…

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Children with Worsening JIA More Likely to Have Uveitis Flares

Children with Worsening JIA More Likely to Have Uveitis Flares

According to new research, children with  juvenile idiopathic arthritis (JIA) are more likely to experience uveitis flares as their arthritis progresses. Medscape notes that researchers wanted to understand how and…

Continue Reading Children with Worsening JIA More Likely to Have Uveitis Flares
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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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