Updates

April 22, 2021April 22, 2021
FDA Puts Hold on KalVista’s Phase 2 Trial of KVD824 for HAE Attacks

The U.S. Food and Drug Administration (FDA) has placed a hold on KalVista Pharmaceuticals’ proposed Phase 2 trial of KVD824 — which the developer had expected ... Read more
April 19, 2021April 19, 2021
EveryLife Scholarship Fund Open Until May 7 for Adults With Rare Diseases in US

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through ... Read more
April 15, 2021April 15, 2021
Orladeyo Now Reimbursable in Japan via National Health Insurance

Japan’s National Health Insurance System (NHI) has approved the addition of Orladeyo (berotralstat), an oral preventive treatment for hereditary angioedema (HAE) attacks, to its drug ... Read more
April 12, 2021April 12, 2021
Life Quality Gains Evident With Newer Preventive HAE Therapies, Survey Finds

Newer preventive therapies for hereditary angioedema (HAE) lead to a better quality of life and fewer attacks than on-demand treatments, according to a large patient ... Read more
April 8, 2021April 8, 2021
Takhzyro Found More Effective Than Cinryze at Preventing HAE Attacks

Takhzyro (lanadelumab) is more effective than Cinryze at preventing swelling attacks in people with hereditary angioedema (HAE), a new study indicates. Analyses suggested that treatment ... 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

FDA Puts Hold on KalVista’s Phase 2 Trial of KVD824 for HAE Attacks

EveryLife Scholarship Fund Open Until May 7 for Adults With Rare Diseases in US

Orladeyo Now Reimbursable in Japan via National Health Insurance

Life Quality Gains Evident With Newer Preventive HAE Therapies, Survey Finds

Takhzyro Found More Effective Than Cinryze at Preventing HAE Attacks

Ionis’ HAE Therapy Greatly Reduced Monthly Swelling Attacks During Trial

Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds

Haegarda Available for Eligible Patients in Canada Starting April 6

Off-label C1 Inhibitor Concentrate May Be Effective in ACEI-induced Acute Angioedema

Intellia Seeks to Advance Its Gene-editing Therapy to Human Trials

Orladeyo Now Available in France Via Early Access Program

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Takeda Seeks Approval in Japan of Takhzyro to Prevent Swelling Attacks

First Patient Dosed in Phase 2 Trial Testing PHVS416 for HAE Attacks

Pink, Green, Blue and Purple Lights Illuminated World for Rare Disease Day

BioCryst Seeks UK Approval of Orladeyo for Preventing HAE Attacks

Long-term Haegarda Found to Improve Quality of Life in HAE Patients

Orladeyo Recommended in EU as 1st Oral Treatment to Prevent HAE Attacks

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

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

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