According to a recent article from Pharmacy Times, Octapharma’s cutaquig has been approved by the FDA to treat patients who are 2 years of age or older with primary humoral…
In 1960, the FDA approved chlorthalidone for hypertension (high blood pressure). However, doctors were unsure whether this therapy could benefit patients with advanced chronic kidney disease (CKD) who had high…
According to an article from the National Hemophilia Foundation, CSL Behring and uniQure have recently provided an update on their phase III HOPE-B trial. This study is evaluating etranacogene dezaparvovec,…
Recently, Kezar Life Sciences Inc. ("Kezar") released interim data and preliminary results from part two of the Phase 2 MISSION clinical trial. According to BioPharma Journal, the results highlight the…
On December 10, 2021, biotechnology company Active Biotech shared that the first subject was dosed within a Phase 1 clinical trial evaluating laquinimod for non-infectious non-anterior uveitis. What is particularly…
In a recent news release, biopharmaceutical company Aligos Therapeutics, Inc. ("Aligos") shared that the first healthy volunteers were dosed in a Phase 1 clinical trial evaluating ALG-055009 for nonalcoholic steatohepatitis.…
According to a recent article from Physician’s Weekly, two trials recently published are showing the results of striving to improve the management and outcomes in nonalcoholic steatohepatitis (NASH) and…
In 2019, Connor Dobbyn, now 13, was diagnosed with Sanfilippo syndrome type C, also known as mucopolysaccharidosis III (MPS III). After his diagnosis, his parents came to recognize that there…
European patients with recurrent or advanced endometrial carcinoma have a new treatment option, as the European Commission (EC) has approved KEYTRUDA plus LENVIMA. Approval is specifically for adult patients whose…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. For the purpose of this status, "rare"…
According to a news release, researchers from biopharmaceutical company HUTCHMED and pharmaceutical company AstraZeneca launched, at the onset of November 2021, the Phase 3 SAMETA clinical trial. During the trial,…
In mid-November 2021, global biopharmaceutical company Zai Lab Limited ("Zai Lab") shared via news release that the first patient was dosed in the Greater China portion of the ADHERE study.…
According to a late October 2021 news release from biopharmaceutical company Mereo BioPharma Group plc ("Mereo"), the company's therapeutic candidate alvelestat received Orphan Drug designation from the FDA. The therapy…
Recently the Kidney Cancer Association held its International Kidney Cancer Symposium, where medical professionals and other industry members come together to share ideas and push for innovative research and treatments…
Orphan Drug designation in the United States is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions, defined as those affecting under 200,000 Americans.…
It is always exciting to see the onset of a new clinical trial designed to research therapies to fill unmet needs for patients. According to a news release from biotechnology…
On October 18, 2021, biopharmaceutical company Paratek Pharmaceuticals, Inc. ("Paratek") shared via news release that the first patient had enrolled in a Phase 2b clinical trial. Within the trial, researchers…
Clinical trials are fantastic ways to develop a deeper understanding of a specific disease, as well as determine the safety, efficacy, and tolerability of potential treatments. Unfortunately, these treatments are…
According to an InBrief PDF from biotechnology and RNA-targeted therapeutics company Ionis Pharmaceuticals ("Ionis"), topline data from a Phase 2 clinical trial highlighted the benefits of cimdelirsen (IONIS-GHR-LRx) for patients…
Canavan disease is an ultra-rare condition that currently has no FDA-approved therapies, meaning that patients face a severe unmet medical need. BridgeBio Pharma aims to change this with their investigational…
In an October 19 news release from biopharmaceutical company Dicerna Pharmaceuticals, Inc. ("Dicerna"), the company shared results from the Phase 1 PHYOX4 clinical trial. During the trial, researchers sought to…
Let us begin this article recently published in MedCity News by starting with the ending: in order to help millions of rare disease patients waiting for rare disease treatments, a…
According to a news release from biopharmaceutical company Clearside Biomedical, Inc. ("Clearside") and eye health business Bausch + Lomb, a revolutionary treatment called XIPERE (triamcinolone acetonide injectable suspension) was recently…
According to an article recently published in WhoTrades, Dupixent has recently been assessed in a clinical trial for the treatment of uncontrolled prurigo nodularis in adult patients. The data were…
In the past, psoriatic arthritis (PsA) has been relatively difficult to manage or control with current therapies such as biologic disease-modifying anti-rheumatic drugs (bDMARDs). While some patients respond well to…
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