September 19, 2019
Preventative Haegarda Therapy Quickly Reduces Number of HAE Attacks, Trial Analysis Shows
News
Prophylactic, or preventive treatment with Haegarda quickly reduced the number of attacks — sudden episodes of swelling — in ... Read more
September 12, 2019
Pharvaris Raises $66M to Advance Oral Therapy for Hereditary Angioedema
News
Pharvaris reported raising $66 million in a second round of financing, allowing the company to advance the clinical development ... Read more
September 5, 2019
Study Supports Genetic Testing to Diagnose HAE of Unknown Cause
News
Use of genetic testing enabled the detection of hereditary angioedema (HAE) in a previously misdiagnosed woman, found to be ... Read more
September 2, 2019August 30, 2019
‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases
News
Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as ... Read more
August 29, 2019August 29, 2019
Firazyr Safely Treats Acute Attacks in Older Angiodema Patients, Study Reports
News
Older patients with hereditary angioedema types 1 or 2 can as safely be treated for an acute attack with Firazyr (icatibant) ... Read more
August 28, 2019
At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing
News
Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert ... Read more
August 22, 2019August 22, 2019
New Rat Model of HAE More Closely Mimics Human Disease
News
Researchers have developed a new rat model of hereditary angioedema (HAE) that more closely mimics the swelling episodes experienced ... Read more
August 20, 2019August 19, 2019
Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases
News
Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you ... Read more
August 8, 2019
Firazyr of Limited Help with Angioedema Caused by ACE Inhibitors, Pooled Trial Data Suggest
News
Firazyr (icatibant acetate) does not appear to be superior to placebo or symptomatic therapies in treating attacks of angioedema ... Read more
August 2, 2019August 1, 2019
Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card
News
Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks ... Read more
August 1, 2019
Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate
News
Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has ... Read more
August 1, 2019
Regenxbio to Develop Antibody-based Gene Therapy for Hereditary Angioedema
News
Regenxbio is exploring a new hereditary angioedema (HAE) treatment that harnesses viral vectors to deliver an antibody-based gene therapy, ... Read more

September 19, 2019

Preventative Haegarda Therapy Quickly Reduces Number of HAE Attacks, Trial Analysis Shows

News

Prophylactic, or preventive treatment with Haegarda quickly reduced the number of attacks — sudden episodes of swelling — in people with hereditary angioedema (HAE) caused by C1-inhibitor deficiency, an ... Read more

September 12, 2019

Pharvaris Raises $66M to Advance Oral Therapy for Hereditary Angioedema

News

Pharvaris reported raising $66 million in a second round of financing, allowing the company to advance the clinical development of PHA121, its lead investigational oral therapy for hereditary angioedema (HAE). ... Read more

September 5, 2019

Study Supports Genetic Testing to Diagnose HAE of Unknown Cause

News

Use of genetic testing enabled the detection of hereditary angioedema (HAE) in a previously misdiagnosed woman, found to be carrying a gene variant linked with HAE of unknown genetic ... Read more

September 2, 2019August 30, 2019

‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases

News

Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest. Two entries in November’s “Disorder: ... Read more

August 29, 2019August 29, 2019

Firazyr Safely Treats Acute Attacks in Older Angiodema Patients, Study Reports

News

Older patients with hereditary angioedema types 1 or 2 can as safely be treated for an acute attack with Firazyr (icatibant) as younger ones, a study found, even though people 65 ... Read more

August 28, 2019

At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing

News

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such ... Read more

August 22, 2019August 22, 2019

New Rat Model of HAE More Closely Mimics Human Disease

News

Researchers have developed a new rat model of hereditary angioedema (HAE) that more closely mimics the swelling episodes experienced by humans, which might improve studies about the mechanisms that ... Read more

August 20, 2019August 19, 2019

Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases

News

Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people ... Read more

August 8, 2019

Firazyr of Limited Help with Angioedema Caused by ACE Inhibitors, Pooled Trial Data Suggest

News

Firazyr (icatibant acetate) does not appear to be superior to placebo or symptomatic therapies in treating attacks of angioedema (swelling) caused by angiotensin-converting enzyme (ACE) inhibitors, a pooled analysis ... Read more

August 2, 2019August 1, 2019

Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card

News

Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage ... Read more