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.
While gene therapy is a burgeoning technique to treat a number of rare genetic conditions, there are still a few complexities associated with this treatment option. How safe and effective…
Over the next 2-2.5 years, biotech spin-out company Augustine Therapeutics will be working to understand both the underlying biology of Charcot-Marie-Tooth disease (CMT) and potential therapeutic options for patients.…
For over 15 years, researchers and scientists have been working to understand the underlying genetics of macular telangiectasia (MacTel), a rare and debilitating eye disease, through the MacTel Project.…
Despite the pandemic, there were many strides in the field of medicine and research in 2020. For example, researchers first discovered and identified a rare disease called VEXAS syndrome. The…
Recently, the Endocrine Society's virtual Annual Meeting, dubbed ENDO 2021, took place from March 20 - 23, 2021. During the meeting, attendees discussed the latest in endocrinology research, treatment,…
At the Endocrine Society's Annual Meeting, or ENDO 2021, Amolyt Pharma ("Amolyt") shared positive clinical data from a Phase 1 clinical trial evaluating AZP-3601 for patients with hypoparathyroidism. Although…
In a press release from early March 2021, cell therapy company CiRC Biosciences, Inc. ("CiRC") shared that its chemically induced photoreceptor-like cells (CiPCs) received Orphan Drug designation from the FDA.…
124 million cases worldwide, with 2.73 million associated deaths. These are the numbers for COVID-19, a novel coronavirus pandemic which, over the past 12-15 months, has swept the globe. In the…
During the Society of Gynecological Oncology 2021 Virtual Annual Meeting on Women’s Cancer, researchers presented data from the Phase 2 OVARIO clinical trial. According to Cancer Network, the trial evaluated…
An estimated 13-20 out of every 100,000 people around the globe have idiopathic pulmonary fibrosis (IPF). The condition often has a poor prognosis (3-5 years following diagnosis) and there are…
Rocket Pharmaceuticals, Inc. ("Rocket") has dedicated its mission to developing gene therapy solutions for rare childhood conditions. Recently, the company shared the publication of preliminary data from a Phase…
In a recent press release, biopharmaceutical company Aeterna Zentaris Inc. shared that the company would be developing a new therapy for patients with hypoparathyroidism. The preclinical development program will focus…
Genomic testing is a great way to learn not just about your genes, but how your genes impact your health. In support of the Phase 2 CRESTONE clinical trial, a…
In a press release from late February 2021, biopharmaceutical company Retrotope shared that its lead drug candidate, RT001, received Rare Pediatric Disease designation for the treatment of patients with Friedreich's…
In late March 2021, the Hypersomnia Foundation and biopharmaceutical company Jazz Pharmaceuticals, Inc. ("Jazz") partnered up to launch the "I Have IH" campaign. The campaign is designed to raise awareness…
The drug development process is not always easy or quick. It consists of drug discovery, preclinical and clinical research, and FDA approval. However, sometimes developers are sent back to the…
How much should medication cost, and are these treatments cost-effective and accessible for patients? In a press release, biopharmaceutical company Aurinia Pharmaceuticals, Inc. ("Aurinia") shared that an independent review performed…
Since its inception, life sciences company Solid Biosciences Inc. has been working to develop treatment options for patients with Duchenne muscular dystrophy (DMD). According to a recent press release, the…
In a recent press release, biopharmaceutical company Swedish Orphan Biovitrum AB ("Sobi") shared that the first patient was dosed in a Phase 3 clinical trial evaluating avatrombopag for pediatric patients…
In March 2021, the FDA expanded access approval to biotechnology company Longeveron Inc.'s investigational Lomecel-B to a pediatric patient with hypoplastic left heart syndrome (HLHS). According to a press…
Together, biopharmaceutical company Sanofi and biotechnology company Regeneron Pharmaceuticals, Inc. ("Regeneron") have been working to develop Libtayo (cemiplimab) for patients with advanced cervical cancer. According to a press release,…
Since its conception, biotechnology company Chinook Therapeutics, Inc. ("Chinook") has worked to develop therapeutic solutions for patients with kidney diseases. Now, the company enrolled the first patient in a…
During the drug development process, multiple studies are performed to evaluate efficacy, safety, tolerability, pharmacokinetics, and other factors. One of these studies is called a multiple ascending dose study, during which…
For nearly 130 years, pharmaceutical company Merck & Co ("Merck" or "MSD") has been working to develop unique therapeutic solutions for those with rare cancers and a variety of…
Fast track designation is granted by the FDA. If a drug developer receives this designation, they begin a process to develop and expedite review for drugs or biologics which treat…
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