Updates

February 26, 2021February 26, 2021
Single PHA121 Dose Can Block Bradykinin Effects in Healthy Volunteers

A single dose of PHA121 (PHA-022121), Pharvaris’ investigational therapy for hereditary angioedema (HAE), is safe and can rapidly and sustainably block bradykinin-induced changes in blood ... Read more
February 25, 2021February 25, 2021
Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a ... Read more
February 24, 2021February 23, 2021
Rare Disease Day at NIH, Set for March 1, Growing Year by Year

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research ... Read more
February 22, 2021February 22, 2021
C1 Inhibitor Use May Safely Prevent HAE Attacks in Pregnant Women

Subcutaneous (under-the-skin) administration of human plasma-derived C1 inhibitor is an effective and safe option for managing hereditary angioedema during pregnancy and lactation, a case report ... Read more
February 19, 2021February 19, 2021
HAE Patients in US Favored Oral Prophylactic in 2018 Survey

People with hereditary angioedema (HAE) would prefer an oral medication that could prevent their attacks, even when satisfied with their current treatment, a survey of ... 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, “A ... Read more

Updates

Single PHA121 Dose Can Block Bradykinin Effects in Healthy Volunteers

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

C1 Inhibitor Use May Safely Prevent HAE Attacks in Pregnant Women

HAE Patients in US Favored Oral Prophylactic in 2018 Survey

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

Stress Can Affect Patients’ Mental Health, How They Manage Their Care

On-demand KVD900 Speeds Symptom Relief, Reduces Use of Rescue Medicines

Pandemic Won’t Stop Rare Disease Day on Feb. 28

Menopause Found to Not Worsen HAE Severity in Most Women

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

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Catabasis Using $110M Investment to Advance QLS-215 Therapy

Sandoz Brings New Generic for Firazyr, Treating Acute HAE Attacks, to US

PHA121, Oral Therapy for HAE Attacks, Safe at All Doses in Phase 1 Trial

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

Orladeyo Approved in Japan to Prevent Swelling Attacks Due to HAE

Rare Genetic Variations in KNG1, XPNPEP1 Linked to HAE With Normal C1-INH

NORD’s Caregiver Respite Program Continues Through Pandemic

Art Contest Salutes Creativity of Rare Disease Communities

COVID-19 Inflammation May Result in Angioedema Symptoms, Case Report Finds

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

What Do Most HAE Patients Want From Treatment? Fewer Attacks, Survey Finds

3 Methods for Measuring C1-INH Activity Give Comparable Results

New Ruconest Manufacturing Facility Will Be Built in 2021

Just-approved Orladeyo Now Being Shipped to US Patients

Takhzyro Approved in China for Routine Prevention of HAE Attacks

Oral PHA121 May Be Better On-demand HAE Treatment Than Firazyr, Early Data Show

FDA Approves Orladeyo, 1st Oral Treatment to Prevent HAE Attacks

New Generic Version of Firazyr Now Available in US to Treat Acute HAE Attacks

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

Injectable Preventive Treatments Weigh on Patients and Caregivers, Surveys Show

Takhzyro Safely Prevents HAE Attacks Over Long Term, Extension Study Shows

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

Single PHA121 Dose Can Block Bradykinin Effects in Healthy Volunteers

Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion

Rare Disease Day at NIH, Set for March 1, Growing Year by Year

C1 Inhibitor Use May Safely Prevent HAE Attacks in Pregnant Women

HAE Patients in US Favored Oral Prophylactic in 2018 Survey

Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients

Stress Can Affect Patients’ Mental Health, How They Manage Their Care

On-demand KVD900 Speeds Symptom Relief, Reduces Use of Rescue Medicines

Pandemic Won’t Stop Rare Disease Day on Feb. 28

Menopause Found to Not Worsen HAE Severity in Most Women

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

Subscribe to our mailing list

Catabasis Using $110M Investment to Advance QLS-215 Therapy

Sandoz Brings New Generic for Firazyr, Treating Acute HAE Attacks, to US

PHA121, Oral Therapy for HAE Attacks, Safe at All Doses in Phase 1 Trial

COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say

Orladeyo Approved in Japan to Prevent Swelling Attacks Due to HAE

Rare Genetic Variations in KNG1, XPNPEP1 Linked to HAE With Normal C1-INH

NORD’s Caregiver Respite Program Continues Through Pandemic

Art Contest Salutes Creativity of Rare Disease Communities

Better Quality of Life Evident in HAE Patients Using Takhzyro in Pivotal Trial

COVID-19 Inflammation May Result in Angioedema Symptoms, Case Report Finds

NORD Seeks Speakers for 2021 Virtual ‘Living Rare, Living Stronger’ Forum

What Do Most HAE Patients Want From Treatment? Fewer Attacks, Survey Finds

3 Methods for Measuring C1-INH Activity Give Comparable Results

New Ruconest Manufacturing Facility Will Be Built in 2021

Just-approved Orladeyo Now Being Shipped to US Patients

Takhzyro Approved in China for Routine Prevention of HAE Attacks

Oral PHA121 May Be Better On-demand HAE Treatment Than Firazyr, Early Data Show

FDA Approves Orladeyo, 1st Oral Treatment to Prevent HAE Attacks

New Generic Version of Firazyr Now Available in US to Treat Acute HAE Attacks

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

Injectable Preventive Treatments Weigh on Patients and Caregivers, Surveys Show

Takhzyro Safely Prevents HAE Attacks Over Long Term, Extension Study Shows

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