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.
Often misdiagnosed as other conditions such as Rett syndrome or cerebral palsy, DDX3X mutations cause both cognitive and physical difficulties. Since only an estimated 700 patients have been diagnosed…
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…
Unfortunately, many molecules which drive cancer development and growth are considered "undruggable." In short, this means that drugs are unable to bind to or address these molecules due to size,…
Even though my boyfriend's mom works at an eye doctor, I'm still kind of afraid to get eye exams - and I'm not alone. Unfortunately, most people do not receive frequent enough…
The European Hematology Association (EHA) held its 2021 Virtual Congress from June 9-17, 2021, though the insights will be available until mid-August 2021. During the Congress, researchers discussed new…
Dr. Jerry Vockley, MD, PhD, who currently works as director of the Center for Rare Disease Therapy at UPMC Children's Hospital of Pittsburgh, was confounded. Through his research, Dr. Vockley…
It's no secret that illicit substance use, or substance use disorders, can cause a variety of health effects, including increased risks for certain diseases. As Parkinson's News Today recently reported,…
In patients with rare or underserved conditions, it can sometimes be difficult to receive effective treatment. For example, chemotherapy, the standard-of-care for Langerhans cell histiocytosis (LCH), is only effective in…
Partnerships within the medical community play a huge role in advancing research and developing treatments. According to Charcot-Marie-Tooth News, a new partnership has formed between Dr. Alessandra Bolino, PhD, the…
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…
In some cases, serious complications occur following allogeneic stem cell or bone marrow transplants. For example, in patients with Graft-versus-host disease (GvHD), the donor cells may recognize patient cells as…
According to Muscular Dystrophy News, new research suggests that the cholesterol metabolic pathway could offer a potential therapeutic target for patients with Duchenne muscular dystrophy (DMD). Following microRNA analysis, researchers…
Within the United States, the FDA developed the Orphan Drug Act to incentivize companies to develop drugs and biologics intended to treat patients with rare conditions. In this case, "rare"…
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…
Anybody who knows me knows that Jeopardy! is one of my favorite shows (Rest In Peace, Alex Trebek). Of course, that doesn't mean that the show never messes up or makes…
Many faced with a rare disease or condition often experience financial burdens in seeking out adequate educational tools, therapeutic devices, medical equipment, resources, treatment options, and more. As described by…
Yeast can be used in baking, brewing, and fermenting. But could it also be used to treat patients with inflammatory bowel disease (IBD)? According to Medical XPress, doctors believe so.…
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…
Genome editing is a topic which has been on the forefront of medical discussion for years. After all, gene editing allows for the potential to treat - or even cure…
CRISPR is a gene-editing technology which holds the potential to treat a variety of genetic diseases and improve patient outcomes. Overall, CRISPR works by creating precise cuts and edits within…
In a news release from June 29, 2021, biotechnology company Neurogene Inc. shared that its adeno-associated virus (AAV) encoding a codon-optimized human CLN5 transgene (hCLN5) received Orphan Drug designation from…
An Orphan Drug designation is granted to drugs or biologics intended to treat patients with rare diseases or conditions, affecting less than 200,000 Americans. Because it can sometimes be difficult…
In the past, antipsychotics were recommended as a potential treatment for patients with Parkinson's disease (PD). This is because, in later stages of PD, patients may experience Parkinson's psychosis, which…
From June 4-8, 2021, the American Society of Clinical Oncology (ASCO) held its Annual Meeting to discuss trends and research within the oncology field. According to Cancer Therapy Advisor,…
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…
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