In a recent press release, biopharmaceutical company AstraZeneca announced that its drug CALQUENCE (acalabrutinib) met both its primary and secondary endpoints in the Phase 3 ELEVATE-RR clinical trial. These endpoints…
On February 8, 2021, global biopharmaceutical company Bristol Myers Squibb shared that the European Commission (EC) approved a Marketing Authorization Application (MAA) for Inrebic (fedratinib). For around 10 years, there…
In a recent press release, biopharmaceutical company Mirum Pharmaceuticals, Inc. ("Mirum") shared that the company finished its rolling New Drug Application (NDA) submission. The NDA focuses around maralixibat, an investigational…
According to a recent press release, pharmaceutical company Karyopharm Therapeutics Inc. ("Karyopharm") achieved a milestone in the NEXPOVIO (selinexor) drug development process. The Committee for Medicinal Products for Human Use…
In a news release from biopharmaceutical company Bristol Myers Squibb, the company shared that the FDA accepted its supplemental New Drug Application (sNDA). Because the sNDA was submitted alongside…
On January 13, 2021, pharmaceutical company Marinus Pharmaceuticals, Inc. ("Marinus") shared via press release that the FDA gave the company a positive response following efficacy and safety data from the…
Research has shown that there is a relationship between type 2 diabetes and cardiovascular disease, an umbrella of diseases that include damaged blood vessels, blood clots, and other issues. In…
Much of our news in 2020 was filled with headlines around COVID-19, the viral pandemic which now has over 96.2 million global diagnoses. As we enter into 2021, the numbers…
According to a study on hindawi.com, the presence of abnormally elevated levels of serum IgG4 was correlated with a greater likelihood of patients living with autoimmune hepatitis to present with…
In an exciting press release, gene therapy platform company Lysogene announced that it had received approval from the Medicines and Healthcare products Regulatory Agency (MHRA) and Research Ethics Committee. As…
In early January 2021, biopharmaceutical company BlueRock Therapeutics ("BlueRock"), in collaboration with the Memorial Sloan Kettering Cancer Center, announced via press release that its Investigational New Drug (IND) application for…
BPD Bronchopulmonary dysplasia (BPD) is a rare condition that primarily affects the lungs of premature babies (though it can affect full term newborns as well). Most children with BPD are born…
According to Parkinson's News Today, the first patient has been dosed in a Phase 1 clinical trial run by IMAC Holdings. The clinical trial is designed to evaluate mesenchymal…
In a recent press release, biotechnology company VISEN Pharmaceuticals ("VISEN") announced the approval of its Investigational New Drug application (IND) in China. The China Center for Drug Evaluation (CDE) accepted…
For the last year, COVID-19 has dominated the headlines. The viral pandemic, present in over 200 countries and territories across the globe, is responsible for 86.7 million cases worldwide,…
According to a recent press release, biopharmaceutical company Kedrion Biopharma ("Kedrion") achieved an important milestone in its Phase 3 CARES10 trial: the last treated patient. During the CARES10 trial, researchers evaluated…
In early December 2020, many descended upon the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition to discuss insights in the field of hematology. During the Meeting, researchers…
According to European Pharmaceutical Review, results from a Phase 1/2a clinical trial support the use of AstroRx for patients with amyotrophic lateral sclerosis (ALS). The clinical trial, headed by Kadimastem…
In a recent press release, biopharmaceutical company Rhythm Pharmaceuticals (“Rhythm”) announced positive data from a Phase 3 clinical trial. During the trial, Rhythm evaluated setmelanotide for patients with Bardet-Biedl syndrome…
Recently, Business Insider reported on published, peer-reviewed results following the Adaptive COVID-19 Treatment Trial (ACTT-2). The Phase 3 clinical trial, sponsored by the National Institute of Allergy and Infectious Diseases…
In a recent press release, Kite, a biopharmaceutical company that is part of Gilead Sciences, announced one-year follow-up results from the ZUMA-2 clinical trial. Within the trial, researchers evaluated Tecartus…
Since its conception, biopharmaceutical company Geron Corporation ("Geron") has worked to develop first-in-class therapies for hematologic myeloid malignancies. More recently, Geron began evaluating imetelstat for patients with lower risk myelodysplastic syndromes…
In a recent press release, biopharmaceutical company Acceleron Pharma, Inc. ("Acceleron") announced that its investigational drug candidate sotatercept received Orphan designation from the European Commission (EC) for the treatment of…
In early December 2020, mRNA medicines company Arcturus Therapeutics Holdings Inc. ("Arcturus") announced that the company begun dosing participants within its Phase 1b clinical trial. Within the trial, researchers will…
In early December, biotechnology company Aeglea BioTherapeutics, Inc. ("Aeglea") announced that its therapy ACN00177 received Rare Pediatric Disease designation from the FDA for the treatment of homocystinuria. Should this drug…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
