Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.
Sometimes, clinical trials do not go the exact way that is expected. According to Targeted Oncology, this is what happened in a Phase 1b clinical trial evaluating RVU120 for…
Unfortunately, there are a great deal of inequities within the healthcare system. Health inequity occurs when groups are treated differently, offered different or worse care, or have a lower…
According to a press release from July 15, 2021, the Chinese NMPA recently approved an Investigational New Drug (IND) application submitted for CAN106, a potential therapeutic option for patients with…
Near the end of June 2021, pharmaceutical company Crinetics Pharmaceuticals, Inc. ("Crinetics") shared via news release that the first patient had been dosed in the Phase 3 PATHFNDR-1 clinical trial.…
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…
In the past, researchers have shown that acute myeloid leukemia (AML) may be driven by certain genetic mutations. For example, some mutations might cause the abnormal or excess production of…
Genetic editing has the potential to address and treat a variety of genetic conditions. Recently, researchers explored gene editing as a potential therapeutic option for patients with facioscapulohumeral muscular dystrophy…
At the end of June 2021, pharmaceutical company Harmony Biosciences Holdings, Inc. ("Harmony") shared the initiation of a Phase 2 clinical trial via news release . During the trial, the…
In the European Union (EU), Orphan Drug designation is granted by the European Medicines Agency (EMA). The status is given to drugs or biologics intended to treat, prevent, or diagnose…
In the United States, the Orphan Drug Act was developed to expedite the development and review of drugs or biologics intended to treat rare or life-threatening conditions. For the purpose…
Have you ever heard of Bachmann-Bupp Syndrome (BABS)? If not, don't worry, you're not alone. In fact, BABS was only first discovered over the past few years. According to Medical…
If you could be vaccinated against serious conditions via nose drops, would you take that option? Well, it could one day be a possibility. According to New Atlas, scientists from…
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…
In the past, Crysvita was approved for subcutaneous administration by a physician for patients with X-linked hypophosphatemia (XLH). This injection could be both invasive and time-consuming. However, according to 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…
Even though some treatments are beneficial for patients, these same treatments can also cause adverse reactions. This seems to be the case for Isturisa (osilodrostat), according to Cushing's Disease News.…
From July 2-3 and 8-10, 2021, the European Crohn's and Colitis Organisation (ECCO) held its 2021 virtual Annual Congress (ECCO'21). During the Congress, many discussed new research and trends…
The healthcare system is designed to provide safe, effective, and beneficial care for all. Unfortunately, the current system represents a wealth of socioeconomic and racial disparities. Past studies have shown…
Seven years ago, when Hunter Parrick was 10 years old, he went for what could've been a routine ear surgery. However, during the surgery, doctors found out that Hunter was…
For patients with Cushing syndrome, transsphenoidal surgery can be a beneficial option for addressing pituitary growths or tumors. But according to Cushing's Disease News, this same surgery also increases the…
In the United Kingdom, the Medicines and Healthcare products Regulatory Agency (MHRA) regulates medication, transfusion components, and medical devices, making sure they meet safety and efficacy standards. According to Pharma…
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 a recent news release from biopharmaceutical company Ultragenyx Pharmaceutical Inc. ("Ultragenyx"), the company's therapy Mepsevii (vestronidase alfa) was approved in Spain for reimbursement. The entire reimbursement process takes…
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