According to a recent news release, CTX001, a gene-edited therapy for patients with transfusion-dependent beta thalassemia, received Priority Medicines (PRIME) designation from the European Medicines Agency (EMA). The treatment was…
Over the last 50 years, doctors have been working to improve clinical outcomes for patients with a rare but aggressive form of ocular cancer: uveal melanoma. However, there has been…
According to the Pharmacy Times, patients with chronic inflammatory demyelinating polyneuropathy (CIDP) now have a new and potentially more effective treatment option. After submitting its supplemental Biologics License Application (sBLA),…
The 2021 virtual American Academy of Neurology (AAN) Annual Meeting is taking place from April 17 through April 22, 2021. During the conference, experts and researchers are offering a…
According to News Medical, researchers from the University of Cincinnati presented findings on a potentially new and effective treatment option for Pompe disease at the virtual American Academy of Neurology…
In a recent press release, RNA-targeted therapeutics company Ionis Pharmaceuticals, Inc. ("Ionis") shared the initiation of a Phase 2/3 clinical trial evaluating ION373 for patients with Alexander disease (AxD). Currently,…
According to a recent news release, the FDA cleared an Investigational New Drug (IND) application for VIP152, a potential therapeutic option for patients with Richter syndrome and relapsed/refractory chronic…
Clinical trials can be beneficial in many ways. For one, these trials show researchers whether a treatment’s impact is durable and sustained. Determining this often requires some follow-up period. According…
On April 1, 2021, biopharmaceutical company AbbVie shared that new data is available from the Phase 3 SELECT-PsA 1 clinical trial. The 24-week results follow the impact of RINVOQ (upadacitinib)…
In late March 2021, the FDA gave Fast Track designation to annamycin. The treatment, initially developed for patients with acute myeloid leukemia (AML), is now being explored for patients with…
In a recent news release, biopharmaceutical company FibroGen, Inc. ("FibroGen") shared that its therapeutic candidate, pamrevlumab, received Fast Track designation from the FDA. The anti-CTGF antibody is designed to treat…
During clinical trials, Independent Data Safety Monitoring Boards (DSMBs) sometimes assess trial safety, trial integrity, and how well the trials are upholding patient needs. According to Business Insider, the DSMB…
From March 19-22,the Society of Gynecological Oncology (SGO) held its 2021 Annual Meeting on Women's Cancer. Because of COVID-19, the event was held virtually. During the meeting, researchers presented…
According to a press release from April 8, 2021, a partnership between Project ALS and the ALS Association has allowed for the development of a Phase 3 clinical trial. Two…
In a recent press release, clinical stage biotech and biopharmaceutical company Biom Therapeutics ("Biom") shared that its drug candidate BIO017 was granted Orphan Drug designation. The treatment, designed for patients…
At the very end of March 2021, specialty pharmaceuticals company JCR Pharmaceuticals Co., Ltd. ("JCR") shared that its investigational drug candidate JR-171 received Orphan Drug designation from the European Medicines…
Financial Burdens in Rare Disease In 2019, 20% of all adults in the United States have reported that they had large medical bills that were unexpected. 18% currently had medical…
Lyme disease, and other tick-borne illnesses, can cause intense and prolonged symptoms. In fact, some patients experience chronic symptoms for a number of years following the initial bite. However,…
On March 29, 2021, global biopharmaceutical company Bristol Myers Squibb ("BMS") shared that its type II variation application for Opdivo (nivolumab) was validated by the European Medicines Agency (EMA).…
In a recent press release, clinical-stage gene therapy company Rocket Pharmaceuticals, Inc. ("Rocket") shared that its investigational gene therapy candidate, RP-L201, received Priority Medicines (PRIME) designation from the European…
During the Society of Gynecological Oncology 2021 Virtual Annual Meeting on Women’s Cancer, researchers presented data from the Phase 2 OVARIO clinical trial. According to Cancer Network, the trial evaluated…
Rocket Pharmaceuticals, Inc. ("Rocket") has dedicated its mission to developing gene therapy solutions for rare childhood conditions. Recently, the company shared the publication of preliminary data from a Phase…
Genomic testing is a great way to learn not just about your genes, but how your genes impact your health. In support of the Phase 2 CRESTONE clinical trial, a…
In a press release from late February 2021, biopharmaceutical company Retrotope shared that its lead drug candidate, RT001, received Rare Pediatric Disease designation for the treatment of patients with Friedreich's…
How much should medication cost, and are these treatments cost-effective and accessible for patients? In a press release, biopharmaceutical company Aurinia Pharmaceuticals, Inc. ("Aurinia") shared that an independent review performed…
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