As reported on PharmaBiz, GENESIS Pharma has announced an exclusive agreement with Otsuka Pharmaceutical Europe Ltd. (OPEL) to distribute and commercialize donidalorsen across 14 Central and Eastern European countries, including…
August 2025 was a landmark month for rare disease innovation, as the U.S. Food and Drug Administration (FDA) granted four historic approvals, each representing a first for their respective conditions,…
A significant advancement for people living with hereditary angioedema (HAE) arrived with the recent FDA approval of Ekterly (sebetralstat), the first oral on-demand therapy designed to treat acute HAE attacks…
KalVista Pharmaceuticals has achieved a significant milestone with the U.S. Food and Drug Administration’s (FDA) approval of its new oral therapy for hereditary angioedema (HAE), marking a breakthrough for patients…
According to a story from Business Wire, the biopharmaceutical company Astria Therapeutics, Inc., recently announced that its experimental therapy navenibart has earned the US Food and Drug Administration's Orphan Drug…
Imagine if you had recurring attacks of severe and unexplained swelling beneath your skin. For individuals living with hereditary angioedema (HAE), a rare inherited disorder, this isn't just a mere…
Last year, the FDA placed a clinical hold on an Investigational New Drug (IND) application for deucrictibant for the on-demand and prophylactic treatment of individuals living with hereditary angioedema…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
Editor's Note: We believe that patients are a key part of developing and leading the conversation in disease communities. Patient Worthy sometimes partners with reputable agencies that wish to speak…
Treatment options such as Haegarda and Takhzyro have significantly improved the treatment landscape for people living with hereditary angioedema (HAE). The treatments, used as prophylaxis, work to reduce disease burden…
In the past, there have been limited therapeutic options for pediatric patients living with hereditary angioedema (HAE). Children aged 2 to <6 years old had no approved prophylaxis therapy. For…
According to a late October 2022 news release from pharmaceutical company KalVista Pharmaceuticals, Inc. (“KalVista”), the company released positive data from a Phase 1 study evaluating oral sebetralstat for…
According to a story from BioPharma Dive, KalVista Pharmaceuticals has halted its clinical trial evaluating an investigational therapy in development for hereditary angioedema. The trial had included a total of…
From October 3-21, 2022, HAE Junior, a Patient Worthy partner organization, will have its art exhibit on display in the halls of the Czech Parliament. HAE Junior is an advocacy…
A study of hereditary angioedema (HAE) patients in Brazil was featured in an article in the Angioedema News. Researchers found that patients waited on average seventeen years for a…
HAE Junior, an advocacy group from the Czech Republic and a Patient Worthy partner organization, is issuing a renewed call for submissions for its Stronger Than HAE art exhibition. This…
Raising awareness is extremely important when it comes to rare diseases. After all, rarity typically results in minimal public and medical awareness. That's why patient communities and other dedicated individuals…
In patients with hereditary angioedema (HAE), the C1-inhibitor blood protein does not function properly, allowing bradykinin peptides to build up in the body. These then cause inflammation and fluid leakage;…
Note: this story was originally published by HAE Junior, a Patient Worthy partner Interview with Malena Vetterli from the European Reference Network (ERN-RITA) for rare immunodeficiencies, autoinflammatory and autoimmune diseases.…
KalVista Pharmaceuticals plans to initiate a Phase 3 trial for KVD900, a treatment for hereditary angioedema (HAE) after finding positive results from earlier Phase 1 and 2 trials. The Phase…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
In the past, there have been little to no concrete studies evaluating the relationship between hereditary angioedema (HAE) and cancer risk. However, researchers questioned whether some sort of relationship could…
In a December 13 press release listed on the company's website, genome editing company Intellia Therapeutics, Inc. ("Intellia") shared that the first patient had been dosed in a Phase…
As parents, we know our son, now fourteen, the best. And my husband and I had expected that at school, they could get to know him well too. We hoped…
Over the last few months, researchers have been enrolling and dosing patients with hereditary angioedema (HAE) from Europe, Israel, and Canada within the Phase 2 RAPIDe-1 clinical trial. According to…
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