According to Newswise, data from a recent clinical trial evaluating IB1001 for patients with Niemann-Pick disease type C (NPC) shows the therapy as effective, safe, and well-tolerated. Sponsored by biopharmaceutical…
Recently, during the virtual 28th United European Gastroenterology Week Congress, the Janssen Pharmaceutical Companies of Johnson & Johnson announced 5-year results from a Phase 3 extension study on STELARA…
While many pharmaceutical companies submit approval and marketing applications, not all are successful. According to Fierce Biotech, a subsidiary of Questex, Santhera Pharmaceuticals ("Santhera"), is one of the most recent…
In the past, there existed no approved treatments for alkaptonuria (AKU), a rare genetic disorder characterized by homogentisate dioxygenase deficiency. However, according to Medical XPress, this recently changed as British…
Near the end of September, Hansa Biopharma ("Hansa") announced promising data from a Phase 2 clinical trial on imlifidase. This therapy is designed to treat patients with severe anti-GBM antibody…
Historically, clinical trials have played an immense role in collecting patient data, understanding disease progression, and crafting more targeted treatments. This is especially true in the oncology realm. But according…
On October 2, 2020, biotechnology company BioMarin Pharmaceutical ("BioMarin") announced that its investigational gene therapy candidate BMN 307 received the Fast Track designation from the FDA. The therapy is designed…
Recently, the Janssen Pharmaceutical Companies of Johnson & Johnson ("Janssen") announced the FDA approval of Simponi Aria (golimumab). The therapy is designed to treat pediatric patients (ages 2 or older)…
Recently, Canada made strides towards better, more accessible treatments for patients with beta thalassemia when they approved REBLOZYL (luspatercept). Biopharmaceutical companies Bristol Myers Squibb Canada ("BMS Canada") and Acceleron Pharma…
According to Medical Xpress, there may be a new treatment option for patients with Duchenne muscular dystrophy (DMD). Traditionally, DMD is treated with glucocorticoids. However, these often require high doses…
Recently, the European Medicines Agency (EMA) recommended Olumiant (baricitinib) for the treatment of adult patients with moderate to severe atopic dermatitis. Currently, Olumiant is approved for the treatment of…
This past week, Bristol Myers Squibb shared four-year data from the Phase 3 CheckMate-214 clinical trial. Ultimately, the trial sought to understand the safety, efficacy, and tolerability of Opdivo…
Last month, the annual ESC Congress, headed by the European Society of Cardiology, discussed innumerable treatments and advances in the field of cardiology. During the conference, biopharmaceutical company Kiniksa Pharmaceuticals…
Although we are now months into the global pandemic, new information emerges daily. Currently, COVID-19 is responsible for over 29 million cases globally, with 926,000 associated deaths. In the United…
There is currently an unmet medical need for therapies and research for Parkinson's disease, with the incidence increasing drastically. In fact, the number of cases around the world is expected…
Early this week, clinical-stage biopharmaceutical company Enterome announced that the first patient was dosed in a Phase 1/2 clinical trial. The SPENCER trial will evaluate EO2401 as a potential treatment…
Between August 29 and September 1, researchers joined in at ESC 2020 - The Digital Experience, developed and organized by the European Society of Cardiology (ESC). During this online conference,…
To determine the safety and efficacy of experimental gene therapy ST-920, researchers first examined the impact of the drug on mouse models of Fabry disease. As reported in Fabry Disease…
Rocket Pharmaceuticals has recently dosed the first patient in their trial of RP-A501, a treatment for Danon disease. According to BioSpace, this trial will test the safety, tolerability, and efficacy…
Recently, biopharmaceutical company Albireo Pharma announced positive results from a Phase 3 clinical trial. The trial, PEDFIC 1, studied the safety, efficacy, and tolerability of odevixibat for patients with progressive…
Earlier this week, the FDA approved oral azacitidine (Onureg) for patients with acute myeloid leukemia (AML). According to the ASCO Post, Onureg continuously treats adult patients who previously achieved remission.…
According to BioSpace, a Phase III trial of Farxiga has produced very positive results. This chronic kidney disease (CKD) treatment was able to dramatically prolong survival in those with stages…
Recently, biopharmaceutical company Fortress Biotech announced positive clinical results for CUTX-101 (copper histidinate). The data highlights the efficacy of CUTX-101 for patients with Menkes disease. As there are currently no…
As reported in OncLive, intravenously-administered rigosertib failed to meet the primary endpoint within the Phase 3 INSPIRE clinical trial. Ultimately, the trial sought to understand whether rigosertib could improve overall…
The 34th Annual Meeting of the Associated Professional Sleep Societies, or "SLEEP 2020," was held last week from August 27th through the 30th. According to BioSpace, pharmaceutical company Harmony Biosciences…
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