June 4, 2020June 4, 2020
Mycophenolate Mofetil May Be Alternative to Cyclophosphamide, Analysis Shows
News
Mycophenolate mofetil (MMF) may be a safe and effective alternative to the standard induction therapy cyclophosphamide in certain ANCA-associated ... Read more
June 1, 2020June 1, 2020
First Part of IFX-1 Trial in AAV Patients in EU Fully Enrolled, InflaRx Says
News
InflaRx announced it has fully enrolled the first part of its Phase 2 trial assessing the safety and efficacy ... Read more
May 28, 2020May 28, 2020
COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say
News, syndicated
While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it ... Read more
May 25, 2020May 25, 2020
Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe
News, syndicated
European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts ... Read more
May 20, 2020
Unity and EU-wide Efforts Focus of Online Rare Disease Meeting
News, syndicated
Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 ... Read more
May 18, 2020May 18, 2020
Request for FDA Approval of Avacopan Likely by Mid-year, ChemoCentryx Says
News
ChemoCentryx is planning to file a request with the U.S. Food and Drug Administration (FDA) by mid-year seeking approval of ... Read more
May 12, 2020May 12, 2020
Rare Diseases Clinical Research Network Opens Online Survey on COVID-19
News, syndicated
The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak ... Read more
May 11, 2020May 11, 2020
Hydralazine Causes Rare Case of AAV with Severe Respiratory, Kidney Failure, Study Reports
News
A common blood pressure medication called hydralazine may cause ANCA-associated vasculitis (AAV), with a severe impact on both ... Read more
May 4, 2020May 4, 2020
AAV Linked to Inflammation, Thickening of Brain Membranes in Case Study
News
A rare case of ANCA-associated vasculitis (AAV) causing thickening of the dura mater — a protective membrane of ... Read more
April 30, 2020April 30, 2020
NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts
syndicated
As the number of Americans infected with COVID-19 surpasses one million, it’s no surprise that people with rare diseases ... Read more
April 29, 2020April 29, 2020
Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak
Announcements
FFF Enterprises and BioNews announced today that both rare and orphan disease advocates are joining forces to provide patients ... Read more
April 29, 2020April 29, 2020
Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say
News, syndicated
While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of ... Read more

June 4, 2020June 4, 2020

Mycophenolate Mofetil May Be Alternative to Cyclophosphamide, Analysis Shows

News

Mycophenolate mofetil (MMF) may be a safe and effective alternative to the standard induction therapy cyclophosphamide in certain ANCA-associated vasculitis (AAV) patients, a review study reports. Based on the data, ... Read more

June 1, 2020June 1, 2020

First Part of IFX-1 Trial in AAV Patients in EU Fully Enrolled, InflaRx Says

News

InflaRx announced it has fully enrolled the first part of its Phase 2 trial assessing the safety and efficacy of IFX-1 in people with ANCA-associated vasculitis (AAV) in Europe. ... Read more

May 28, 2020May 28, 2020

COVID-19 Delaying Rare Disease and Gene Therapy Trials, Pharma Execs Say

News, syndicated

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene ... Read more

May 25, 2020May 25, 2020

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

News, syndicated

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session ... Read more

May 20, 2020

Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

News, syndicated

Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. ... Read more

May 18, 2020May 18, 2020

Request for FDA Approval of Avacopan Likely by Mid-year, ChemoCentryx Says

News

ChemoCentryx is planning to file a request with the U.S. Food and Drug Administration (FDA) by mid-year seeking approval of avacopan (CCX168) as a treatment for people with ANCA-associated vasculitis (AAV), the company ... Read more

May 12, 2020May 12, 2020

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

News, syndicated

The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. ... Read more

May 11, 2020May 11, 2020

Hydralazine Causes Rare Case of AAV with Severe Respiratory, Kidney Failure, Study Reports

News

A common blood pressure medication called hydralazine may cause ANCA-associated vasculitis (AAV), with a severe impact on both lungs and kidneys in rare instances, a case report shows. The ... Read more

May 4, 2020May 4, 2020

AAV Linked to Inflammation, Thickening of Brain Membranes in Case Study

News

A rare case of ANCA-associated vasculitis (AAV) causing thickening of the dura mater — a protective membrane of the central nervous system (CNS) — was found in a person ... Read more

April 30, 2020April 30, 2020

NORD Webinar Outlines COVID-19 Response on Financial, Policy Fronts

syndicated

As the number of Americans infected with COVID-19 surpasses one million, it’s no surprise that people with rare diseases — whose immune systems were generally compromised even before the ... Read more