According to a recent article, a new stem-cell-based gene therapy is showing promising results for treating beta (β)-thalassemia in a phase 1 clinical trial. Beta Thalassemia Beta thalassemia is a…
What if researchers could change DNA processes within brain tumors to improve overall survival rates and patient outcomes? According to Medical XPress, researchers found that L-methylfolate, in conjunction with the…
Clinical trial results can help support drug developers' goals. For example, positive trial results may support Marketing Authorization Applications (MAAs) or other areas needed to get therapies into the…
Back in December, UCB released the results of its phase 3 trial of rozanolixizumab in adult patients with generalized myasthenia gravis (gMG). Fortunately, these data were positive, pointing to this…
Over the last 10+ years, there have been extreme advances in the treatment of metastatic melanoma. In particular, researchers have sought to develop therapies which halted cancer progression and reduced…
Just 50 years ago, receiving a diagnosis of severe aplastic anemia could be devastating. While therapeutic options have greatly improved over the past five decades, some doctors have still found…
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…
In a news release from mid-December 2021, rare liver disease company Albireo Pharma, Inc. ("Albireo") shared that positive topline data was available from a Phase 1 clinical trial evaluating A3907…
According to a December 16, 2021 news release from biopharmaceutical company Applied Therapeutics, Inc., the company has initiated a registrational Phase 2/3 INSPIRE clinical trial to evaluate AT-007 for patients…
According to a news release from RNA therapeutics company ProQR Therapeutics N.V. ("ProQR"), the first patients have been dosed in two Phase 2/3 clinical trials evaluating QR-421a for USH2a-mediated retinitis…
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.…
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