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Trial of Niemann-Pick Disease Treatment is Expanded

Trial of Niemann-Pick Disease Treatment is Expanded

IntraBio has recently launched an extension study of their Phase II trial of IB1001, a treatment for Niemann-Pick disease Type C. This decision was made shortly after analyzing the positive…

Continue Reading Trial of Niemann-Pick Disease Treatment is Expanded
CureVac Joins the Race to Develop a Vaccine Against COVID-19 With Its Successful Phase 1 Trial

CureVac Joins the Race to Develop a Vaccine Against COVID-19 With Its Successful Phase 1 Trial

According to an article in Biospace, CureVac of Boston, MA, with headquarters in Tübingen, Germany, just announced interim results from its Phase 1 study evaluating CVnCoV, a vaccine candidate against…

Continue Reading CureVac Joins the Race to Develop a Vaccine Against COVID-19 With Its Successful Phase 1 Trial
Year Two of This Alport Syndrome Trial Concludes with Encouraging Findings

Year Two of This Alport Syndrome Trial Concludes with Encouraging Findings

According to a story from GlobeNewswire, the biopharmaceutical company Reata Pharmaceuticals, Inc., recently announced that year two of its phase 3 clinical trial has been completed. This trial is testing…

Continue Reading Year Two of This Alport Syndrome Trial Concludes with Encouraging Findings
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…

Continue Reading COVID-19 Vaccine Trial Resumes in the US
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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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