Medical studies are crucial players in the journey to develop new and more efficacious treatment options for patients with rare diseases. On June 10, 2021, Vertex Pharmaceuticals Incorporated ("Vertex") shared…
The FDA has recently granted their approval to a Phase 2 trial of FHL-301, a treatment for those in the earlier stages of Parkinson's disease. Forest Hills Lab will conduct…
According to a news release from late June 2021, biopharmaceutical company Pharming Group N.V. ("Pharming") completed enrollment for a Phase 2/3 clinical trial evaluating leniolisib for patients with activated phosphoinositide…
While myasthenia gravis (MG), an autoimmune neuromuscular disorder, can be treated, there is a smaller subset of patients who are treatment-refractory. Those whose condition is not easily treated are thought…
In the company's Phase 2 proof-of-concept clinical trial, biopharmaceutical company Harbour BioMed ("Harbour") sought to understand the impact of batoclimab (HBM9161) on patients with generalized myasthenia gravis (gMG). The…
Clinical trials can be crucial in determining potential treatment options for a variety of conditions. According to Pharma Biz, biopharmaceutical company Nordic Nanovector ("Nordic") recently shared promising data from the…
Acknowledgment: This story is sponsored by Takeda and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant, vetted and valuable information to the…
Clinical trials are important research tools to help understand the safety, efficacy, and tolerability of potential treatments. According to a news release from July 19, 2021, biotechnology company Prometheus Biosciences,…
According to the Cancer Network, orelabrutinib, a Bruton tyrosine kinase (BTK) inhibitor, was recently granted Breakthrough Therapy designation within the United States. While the treatment is also indicated for patients…
Breakthrough Therapy designation is granted by the FDA as a way to accelerate drug development and review. In particular, the designation is granted to drugs or biologics for serious conditions,…
In a news release from mid-July, 2021, biopharmaceutical company Strongbridge Biopharma plc ("Strongbridge") shared the publication of new post hoc analyses regarding KEVEYIS (dichlorphenamide) for patients with Primary Periodic Paralysis…
Thus far, retifanlimab, a drug designed to treat patients with anal cancer, has received Orphan Drug designation and Priority Review status. However, the approval process has not been entirely easy.…
Unfortunately, clinical trials do not always go the way that researchers intend. Sometimes, issues arise during early or preclinical studies which cause enough concern to stop or pause the…
In the past, arimoclomol has not always shown treatment promise. For example, the drug failed to meet primary or secondary endpoints in a clinical trial evaluating arimoclomol for patients with…
Initially, biopharmaceutical company Anavex Life Sciences ("Anavex") developed Anavex 2-73 (blarcamesine) for patients with Alzheimer's disease. Now, the company has expanded its exploration of the therapy to determine its efficacy…
Clinical trial data is extremely helpful in understanding the safety, efficacy, and tolerability of various potential treatments. According to Muscular Dystrophy News, top-line data from the Phase 2b VISION-DMD…
During the European Hematology Association (EHA)'s 26th Annual Meeting, taking place virtually, biopharmaceutical company Kite (part of Gilead) shared longer-term follow-up data on Yescarta (axicabtagene ciloleucel) for patients with relapsed…
Medical professionals have been searching for a viable treatment for Parkinson's disease for a long time. While there have been advancements and new therapies created, many patients are still looking…
Eosinophilic asthma is a rare and often severe form of asthma which can be difficult to treat. Because of this, many patients with eosinophilic asthma experience diminished quality of life…
In early June 2021, Epidermolysis Bullosa News reported that Filsuvez (Oleogel-S10), a topical gel for epidermolysis bullosa (EB), received Priority Review designation from the FDA. Filsuvez Developed by Amryt Pharma,…
In a news release from June 23, 2021, Revolo Biotherapeutics ("Revolo") shared that the FDA approved an Investigational New Drug (IND) application for the company's '1104 peptide treatment. Altogether, '1104…
According to a relatively recent news release from biopharmaceutical company Applied Therapeutics, Inc., AT-007 received Fast Track designation from the FDA. This therapy, which has also received both Orphan Drug…
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,…
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