For years, researchers have been evaluating pridopidine, developed by Prilenia Therapeutics ("Prilenia"), as a potential therapy for patients with neurodegenerative conditions. As reported in HD Buzz, new research has highlighted…
From June 5-8, 2021, the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) held its 58th Annual Virtual Meeting. Due to COVID-19, the meeting was held fully online. However, that…
In a news release from late May 2021, biopharmaceutical company Timber Pharmaceuticals, Inc. ("Timber") shared that all patients have been enrolled in the Phase 2b CONTROL trial. During the trial,…
Rare Pediatric Disease designation is a status granted by the FDA to drugs or biologics intended to treat rare diseases (affecting under 200,000 Americans) in patients aged 18 or younger.…
Prior to 2021, there were no FDA-approved treatments for patients with type 1 plasminogen deficiency, or hypoplasminogenemia. As a result, there was an urgent need to fill this unmet need…
From June 5-8, 2021, the European Renal Association - European Dialysis and Transplant Association (ERA-EDTA) held its 58th Annual Congress. Though the event was held virtually, the meeting still expanded…
According to a June 11 press release from biotechnology company Equillium, Inc. ("Equillium"), positive topline data is now available from the Phase 1b EQUATE clinical trial. Within the clinical trial,…
Currently, the European Hematology Association (EHA) Virtual Congress 2021 is going on from June 9 through 17, 2021. During the Congress, hematologists and other stakeholders will discuss clinical research and…
Currently, the European Hematology Association (EHA) is holding its 2021 Virtual Congress through June 17, 2021. During the event, researchers are expounding on new insights and research within the hematological…
On June 8, 2021, pharmaceutical and biotechnology company Pfizer Inc. ("Pfizer") shared that its Pneumococcal 20-valent Conjugate Vaccine (PREVNAR 20) is now FDA-approved. The vaccine helps protect against 20 different…
As of June 7, 2021, an injectable treatment is now available for patients aged 1 month+ with paroxysmal nocturnal hemoglobinuria. The FDA recently approved Ultomiris (ravulizumab-cwvz) for these patients. Previously,…
In early June 2021, SPG302, an investigational therapy for patients with amyotrophic lateral sclerosis (ALS), received Orphan Drug designation from the FDA. According to ALS News Today, the treatment…
From June 2-5, 2021, the European Alliance of Associations for Rheumatology (EULAR) held its EULAR 2021 Virtual Congress. During the event, participants discussed basic, translational, and clinical science within the…
According to a recent news release from biopharmaceutical company Arena Pharmaceuticals, Inc. ("Arena"), the company's investigational therapy, etrasimod, received Orphan Drug designation from the FDA. Currently, Arena is evaluating etrasimod…
A recent study investigating treatment for adult acromegaly patients has revealed that switching from injectable somatostatin receptor ligands to oral octreotide leads to an improvement in quality of life and…
Early on in 2021, the FDA approved voclosporin as an add-on treatment for patients with lupus nephritis (LN), a frequent complication of lupus. Ultimately, this approval was a positive decision,…
In a recent news release on Morningstar, biopharmaceutical company Albireo Pharma, Inc. ("Albireo") shared that its investigational drug candidate Bylvay (odevixibat) earned a positive opinion from the European Medicines…
Sometimes, medical studies just don't go the way that companies want them to. According to MedCity News, this is what biotechnology company Larimar Therapeutics ("Larimar") was faced with during…
In a press release from late May 2021, AAV gene therapy company and Bayer AG subsidiary Asklepios BioPharmaceutical, Inc. ("AskBio") shared that the FDA approved its Investigational New Drug (IND)…
From May 11-14, 2021, the American Society of Gene and Cell Therapy (ASGCT) held its 24th Annual Meeting. Due to COVID-19, the meeting was held virtually - but that didn't…
While some therapies offer potential benefits to patients, not all therapies show efficacy within the clinical trial environment. Recently, shares MedCityNews, this occurred with biotechnology company Biogen Inc.'s ("Biogen") gene…
According to Multiple Sclerosis News Today, the European Commission (EC) recently approved PONVORY (ponesimod) for the treatment of adult patients with relapsing and remitting (R/R) or active secondary progressive (SP)…
In a news release from May 19, 2021, startup biotechnology company GeneTx Biotherapeutics LLC ("GeneTx") and biopharmaceutical company Ultragenyx Pharmaceutical Inc. ("Ultragenyx") shared that the pair received Health Canada clearance…
In a news release from May 18, 2021, biopharmaceutical company Catalyst Biosciences, Inc. ("Catalyst") shared that the first patient was dosed in the Phase 1/2 MAA-202 clinical trial. During…
Clinical trials are extremely beneficial in learning how new and burgeoning treatment options can benefit patients. At the same time, these trials also offer insight into where treatments may not…
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