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.
A biological marker ("biomarker") is a measurable trait or characteristic that can tell researchers something about your body or health. For example, body mass index (BMI) is a common biomarker…
On October 2, 2020, biotechnology company BioMarin Pharmaceutical ("BioMarin") announced that its investigational gene therapy candidate BMN 307 received the Fast Track designation from the FDA. The therapy is designed…
Recently, the Janssen Pharmaceutical Companies of Johnson & Johnson ("Janssen") announced the FDA approval of Simponi Aria (golimumab). The therapy is designed to treat pediatric patients (ages 2 or older)…
Recently, Canada made strides towards better, more accessible treatments for patients with beta thalassemia when they approved REBLOZYL (luspatercept). Biopharmaceutical companies Bristol Myers Squibb Canada ("BMS Canada") and Acceleron Pharma…
Recently, the Janssen Pharmaceutical Companies of Johnson & Johnson reported that they submitted a New Drug Application (NDA) for intravenously administered UPTRAVI (selexipag). The therapy is designed to treat…
Developmental and epileptic encephalopathies (DEEs), or rare epilepsies, are somewhat of a newer topic. However, researchers are already making strides in creating treatments to address unmet patient needs. On…
According to Medical Xpress, there may be a new treatment option for patients with Duchenne muscular dystrophy (DMD). Traditionally, DMD is treated with glucocorticoids. However, these often require high doses…
Just last week, biopharmaceutical company Mustang Bio announced Orphan Drug designation for its therapeutic candidate, MB-207. This unique lentiviral gene therapy is designed for previously treated patients with X-linked…
Recently, Blueprint Medicines Corporation ("Blueprint") shared that the European Commission (EC) approved marketing authorization of AYVAKYT (avapritinib). The therapy is designed to treat adult patients with PDGFRA D842V mutated gastrointestinal stromal…
Early last week, biopharmaceutical company MediciNova Inc. ("MediciNova") announced promising results for their therapeutic candidate MN-166 (ibudilast). The company, which published in the findings in Cancer Chemotherapy and Pharmacology, explored…
Last week, biotechnology company Anokion SA ("Anokion") announced the FDA's acceptance of an Investigational New Drug (IND) application. Anokion submitted the IND for ANK-700, a therapy for patients with…
Recently, researchers sought to better understand the natural history and natural progression of type III spinal muscular atrophy (SMA). According to the AJMC, this study offers more insight into…
On September 25, biotechnology company Vertex Pharmaceuticals Incorporated ("Vertex") announced FDA-approval of KALYDECO (ivacaftor) for pediatric patients with cystic fibrosis (CF). Specifically, KALYDECO can treat infants between 4-6 months…
Recently, research and development (R&D) company Neurophth Therapeutics announced that the FDA granted Orphan Drug designation to its therapy NR082. Also known as rAAV2-ND4 or NFS-01 project, NR082 is…
According to a recent press release, Japan just approved ULTOMIRIS (ravulizumab) for patients with atypical hemolytic uremic syndrome (aHUS). Developed by biopharmaceutical company Alexion Pharmaceuticals ("Alexion"), ULTOMIRIS represents a…
Recently, the European Medicines Agency (EMA) recommended Olumiant (baricitinib) for the treatment of adult patients with moderate to severe atopic dermatitis. Currently, Olumiant is approved for the treatment of…
For many years, researchers linked obesity to a number of other health conditions. Now, Taiwanese researchers identify a new cause for concern: worsening health outcomes for patients with ankylosing…
In a recent press release, clinical-stage pharmaceutical company Crinetics Pharmaceuticals announced that their investigational drug candidate, CRN04777, received Rare Pediatric Disease designation from the FDA. The therapy is designed…
In a recent press release, precision therapy company Blueprint Medicines Corporation announced positive results from two clinical trials: EXPLORER and PATHFINDER. The trials examined the safety, efficacy, and tolerability…
Did you know that testing protein levels in our bodies can tell researchers about our health? In some cases, it can even give insight into disease progression and development.…
This year, thanks to COVID-19, many conferences have been held online. These play a crucial role in spreading data, facilitating conversations, and determining potential treatment options for patients with…
I'm sure you have heard about Zika virus before, but what about Chikungunya? Both are rare mosquito-borne diseases that can cause severe neurological problems. But now, researchers also believe…
This past week, Bristol Myers Squibb shared four-year data from the Phase 3 CheckMate-214 clinical trial. Ultimately, the trial sought to understand the safety, efficacy, and tolerability of Opdivo…
Last week, biotechnology company BioMarin announced that Dr. Lynda Polgreen, MD, MS would present at the American Society for Bone and Mineral Research (ASBMR) Annual 2020 Meeting. Her presentation centered…
This past week, Cycle Pharma announced FDA approval of a once daily dose of NITYR for patients with hereditary tyrosinemia type 1 (HT-1). According to its prescribing information, NITYR…
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