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.
According to a recent news release, CTX001, a gene-edited therapy for patients with transfusion-dependent beta thalassemia, received Priority Medicines (PRIME) designation from the European Medicines Agency (EMA). The treatment was…
As Prevention reminds us, tick season is officially upon us! That's right, these little pests enjoy warm and humid weather. So even while we're still social-distancing, ticks are getting ready to show…
Medical research is crucial to expanding an understanding of certain conditions, which ultimately leads to improved diagnostic and treatment options, and more social awareness. In a recent press release, CurePSP…
How effective is guselkumab, a human monoclonal antibody, at treating patients with psoriatic arthritis (PsA)? According to post-hoc data analysis from two clinical trials, the therapy was relatively effective.…
According to a news release from April 2021, a new partnership is working to improve the commercialization of rare disease drugs for patients in China. Together, rare disease therapeutics company…
Peripartum cardiomyopathy is a rare heart ailment, characterized by a weakened heart muscle, that occurs during pregnancy. But why do some pregnant women develop this condition while others do not?…
Over the last 50 years, doctors have been working to improve clinical outcomes for patients with a rare but aggressive form of ocular cancer: uveal melanoma. However, there has been…
In a recent press release, biotechnology and gene therapy company LEXEO Therapeutics ("LEXEO") shared that its gene therapy LX1001 received Fast Track designation from the FDA. Overall, LX1001 is…
If you hear lithium, what's the first thing you think of? For years, lithium has been one of the most widely used and studied therapies for patients with bipolar…
In a news release from March 2021, international pharmaceutical company the Menarini Group shared that it had joined a partnership with Nippon Shinyaku Co., Ltd. ("Nippon") to develop and commercialize…
According to the Pharmacy Times, patients with chronic inflammatory demyelinating polyneuropathy (CIDP) now have a new and potentially more effective treatment option. After submitting its supplemental Biologics License Application (sBLA),…
Over the past 14 or so months, COVID-19 has dominated the headlines. It has reshaped the way that we work, attend school, build relationships with others, and receive medical treatment.…
Did you know that there are over 80 chemicals, called cannabinoids, that can be sourced from the Cannabis sativa plant (marijuana or hemp)? Now, Europe is harnessing this for…
The 2021 virtual American Academy of Neurology (AAN) Annual Meeting is taking place from April 17 through April 22, 2021. During the conference, experts and researchers are offering a…
According to News Medical, researchers from the University of Cincinnati presented findings on a potentially new and effective treatment option for Pompe disease at the virtual American Academy of Neurology…
In a recent press release, RNA-targeted therapeutics company Ionis Pharmaceuticals, Inc. ("Ionis") shared the initiation of a Phase 2/3 clinical trial evaluating ION373 for patients with Alexander disease (AxD). Currently,…
According to a recent news release, the FDA cleared an Investigational New Drug (IND) application for VIP152, a potential therapeutic option for patients with Richter syndrome and relapsed/refractory chronic…
According to HCP Live, a new study may have discovered a connection between severe pediatric atopic dermatitis and learning disabilities. Researchers, spearheaded by Dr. Joy Wan, MD, MSCE from the…
Normally, metformin is used to help control blood sugar in patients with type 2 diabetes. However, new research suggests that the treatment option could also be beneficial for those with…
According to FirstWord Pharma, patients with paroxysmal nocturnal hemoglobinuria (PNH) in the United Kingdom (UK) may soon have a more effective and efficient, and less invasive, treatment option. Currently, the…
In 2009, the FDA approved Tyvaso (treprostinil) Inhalation Solution to reduce symptoms associated with Group 1 pulmonary hypertension (PH), also known as pulmonary arterial hypertension (PAH). Beyond symptom reduction, the…
Growing up, I was always told that oral health related directly to regular health. But now, a study from researchers at the NYU College of Dentistry and Weill Cornell Medicine…
In mid-April, Kantar Health and the International Gaucher Alliance (IGA) shared the launch of a rare disease registry developed by the partnership. The registry, known as GARDIAN, will explore Gaucher…
Earlier this year, WP1066, a p-STAT3 inhibitor, received three separate Rare Pediatric Disease designations for the treatment of medulloblastoma, atypical teratoid rhabdoid tumor, and diffuse intrinsic pontine glioma. Now,…
Clinical trials can be beneficial in many ways. For one, these trials show researchers whether a treatment’s impact is durable and sustained. Determining this often requires some follow-up period. According…
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