Updates

May 25, 2020May 25, 2020
Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

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 ... Read more
May 20, 2020May 20, 2020
Unity and EU-wide Efforts Focus of Online Rare Disease Meeting

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 ... Read more
May 13, 2020May 13, 2020
Costs Unsustainable for New Angioedema Preventive Therapies in US, Real-world Data Show

Costs are unsustainable for preventive treatment of hereditary angioedema (HAE) attacks with approved therapies Takhzyro or Haegarda, according to an analysis of six-month real-world data performed ... Read more
May 12, 2020May 12, 2020
Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

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 ... Read more
May 6, 2020May 6, 2020
European Commission Approves Ruconest for Acute Swelling in Children with HAE

The European Commission has approved Pharming‘s Ruconest (conestat alfa) for the treatment of acute swelling episodes in young children with hereditary angioedema (HAE). This approval ... Read more

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September 27, 2018September 27, 2018

Stroke Patient Develops Orolingual Angioedema After Alteplase Administration, Case Study Reports

News

Researchers from University of Chicago Medicine reported the case of a patient who developed orolingual angioedema after the administration of alteplase (sold under the brand name Activase), and suggest how physicians should proceed in case of an emergency. The study, ... Read more

Updates

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

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

Costs Unsustainable for New Angioedema Preventive Therapies in US, Real-world Data Show

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

European Commission Approves Ruconest for Acute Swelling in Children with HAE

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

Potential Oral Therapy for HAE Seen To Be Effective as Firazyr in Monkey Model

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

EMA to Review Berotralstat as Preventive Treatment for HAE Swelling Attacks

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NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

Berotralstat Lowers Rate of HAE Attacks and Possibly Their Severity, Trial Reports

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

Study Identifies Treatments That Reduce Acute Attacks in HAE Patients With Plasminogen Mutation

Preventive Treatment with Berotralstat Lowers HAE Attack Rate, BioCryst Says

New Production Facility for Ruconest, Pharming’s Acute HAE Treatment, Gets FDA Nod

Preventive Treatment with Berotralstat Lowers HAE Attack Rate, BioCryst Says

New Production Facility for Ruconest, Pharming’s Acute HAE Treatment, Gets FDA Nod

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

Mutations May Raise Risk of Angioedema Linked to Blood Pressure Medicines

Test Using Dried Blood Spot May Be Simple Way to Diagnose HAE

Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29

Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar

FDA Agrees to Review Berotralstat as Preventive Treatment for HAE Attacks

Patients, Supporters Worldwide Recognizing Rare Disease Day 2020

Ruconest Safe, Effective for On-demand and Preventive Treatment of HAE Attacks, Study Says

Study Identifies Novel Molecular Players as Potential Therapeutic Target in Bradykinin-mediated Swelling Attacks

Intellia Presents First Data Supporting CRISPR/Cas9 Gene Therapy Candidate for HAE, Anticipated to Lessen Angioedema

Preventative Therapy for HAE Attacks, Haegarda, Now Available in Quebec

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

Life Expectancy Similar for HAE Patients and General Population in Italy, Study Shows

Stroke Patient Develops Orolingual Angioedema After Alteplase Administration, Case Study Reports

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Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

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

Costs Unsustainable for New Angioedema Preventive Therapies in US, Real-world Data Show

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

European Commission Approves Ruconest for Acute Swelling in Children with HAE

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

Potential Oral Therapy for HAE Seen To Be Effective as Firazyr in Monkey Model

Surge in Telemedicine One ‘Good’ Outcome from COVID-19 Crisis, Doctors Say

Industry leaders seek to advance innovative resources for rare and orphan disease communities amid COVID-19 outbreak

NORD Opens COVID-19 Financial Aid Program for Rare Disease Community

EMA to Review Berotralstat as Preventive Treatment for HAE Swelling Attacks

Subscribe to our mailing list

NORD Partners with ‘Sing Me a Story’ for July 18–20 Living Rare Forum

Berotralstat Lowers Rate of HAE Attacks and Possibly Their Severity, Trial Reports

Global Rare Disease Group’s Focus: 1,000 New Therapies by 2027, Despite COVID-19

Study Identifies Treatments That Reduce Acute Attacks in HAE Patients With Plasminogen Mutation

Preventive Treatment with Berotralstat Lowers HAE Attack Rate, BioCryst Says

New Production Facility for Ruconest, Pharming’s Acute HAE Treatment, Gets FDA Nod

Preventive Treatment with Berotralstat Lowers HAE Attack Rate, BioCryst Says

New Production Facility for Ruconest, Pharming’s Acute HAE Treatment, Gets FDA Nod

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

Video: BioNews’ Social Media Campaign Highlights #WhatMakesMeRare

Mutations May Raise Risk of Angioedema Linked to Blood Pressure Medicines

Test Using Dried Blood Spot May Be Simple Way to Diagnose HAE

Elsevier Opens Books, Review Articles to Rare Disease Researchers Starting Feb. 29

Advice on Starting Nonprofit Groups for Rare and Other Diseases Focus of NORD Webinar

FDA Agrees to Review Berotralstat as Preventive Treatment for HAE Attacks

Patients, Supporters Worldwide Recognizing Rare Disease Day 2020

Ruconest Safe, Effective for On-demand and Preventive Treatment of HAE Attacks, Study Says

Study Identifies Novel Molecular Players as Potential Therapeutic Target in Bradykinin-mediated Swelling Attacks

Intellia Presents First Data Supporting CRISPR/Cas9 Gene Therapy Candidate for HAE, Anticipated to Lessen Angioedema

Preventative Therapy for HAE Attacks, Haegarda, Now Available in Quebec

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

Life Expectancy Similar for HAE Patients and General Population in Italy, Study Shows

Stroke Patient Develops Orolingual Angioedema After Alteplase Administration, Case Study Reports

Pin It on Pinterest