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 an InBrief PDF from biotechnology and RNA-targeted therapeutics company Ionis Pharmaceuticals ("Ionis"), topline data from a Phase 2 clinical trial highlighted the benefits of cimdelirsen (IONIS-GHR-LRx) for patients…
“The patient network is incredibly important,” Lina Williamson, PhD, explained to me. You see, in her childhood, Lina experienced a number of seemingly unexplainable symptoms: severe and sometimes debilitating leg…
In a late October news release from pharmaceutical company Marinus Pharmaceuticals, Inc. ("Marinus"), the company shared that it would be collaborating with the Loulou Foundation, a private UK nonprofit dedicated…
When it comes to advances in hereditary ATTR amyloidosis (hATTR amyloidosis), biopharmaceutical company Alnylam Pharmaceuticals, Inc. ("Alnylam") has been on top of its game. Alnylam helped develop Onpattro and is…
According to Hemophilia News Today, GENV-HEM, an investigational treatment for patients with hemophilia A and B either with or without clotting factor inhibitors, earned Orphan Drug designation from the FDA.…
In the European Union (EU), Orphan Drug designation ("Orphan designation") is granted to products intended to treat, prevent, or diagnose a life-threatening, chronic, or rare disease. These are defined as…
In an October 19 news release from biopharmaceutical company Dicerna Pharmaceuticals, Inc. ("Dicerna"), the company shared results from the Phase 1 PHYOX4 clinical trial. During the trial, researchers sought to…
Sometimes, treatments used in conjunction with each other can confer benefits for patients. According to the Cancer Network, for example, treating platinum-resistant ovarian cancer using nemvaleukin alfa and pembrolizumab (Keytruda)…
While there is no cure for patients with systemic scleroderma, there are some therapies to manage symptoms, such as medications, antibiotics, physical therapy, or even surgery. But according to Medical…
According to a news release from biopharmaceutical company Clearside Biomedical, Inc. ("Clearside") and eye health business Bausch + Lomb, a revolutionary treatment called XIPERE (triamcinolone acetonide injectable suspension) was recently…
When Shelby Sparks was just five months old, she began having grand mal seizures. Throughout her early years, her family just believed that their daughter had severe epilepsy. Then, when…
For years, Debra Papagni Denmark worked to have New York State recognize Phelan-McDermid Syndrome after her son Drew, now 13, was diagnosed. A few years ago, Governor Andrew Cuomo heard…
According to a news article published by the Yale School of Medicine, Yale University researchers recently determined that autosomal dominant polycystic kidney disease (ADPKD) could potentially be reversed. Head researcher…
Also known as Whitmore's disease, melioidosis is a rare and deadly infectious disease. While rare in the United States, melioidosis is considered a public health crisis in northern Australia, Southeast…
When AJ Blinn, now age 12, was just 8 years old, his parents noticed something potentially concerning. As AJ was walking around, he was also limping. Eventually, his parents took…
In 2019, Nathan James Bryan was 31 years old. He enjoyed drinking beer with his friends, working out, and spending time with his family. In fact, he had been staying…
In the past, psoriatic arthritis (PsA) has been relatively difficult to manage or control with current therapies such as biologic disease-modifying anti-rheumatic drugs (bDMARDs). While some patients respond well to…
When a 23-year-old woman, who was 6 weeks pregnant, first appeared at the doctor, she was presenting with non-specific symptoms: pale eyelids, abdominal pain, intermittent cramping, and generalized arm and…
Currently, a glioblastoma multiforme diagnosis comes with a relatively poor prognosis: 25% survival after 1 year, and 5% after 5 years. Researchers have been looking to not only expand their…
From October 6-7, 2021, researchers and stakeholders descended upon the virtual Northeast Amyotrophic Lateral Sclerosis Meeting to discuss new trends, research, and therapies within the field. According to ALS News…
According to a recent news release, the first cohort - consisting of healthy volunteers - was dosed in a Phase 1 clinical trial evaluating an intranasal formulation of 5-MeO-DMT. Ultimately,…
When drug developers receive marketing approval in different countries, they expand medication access and allow patients across the globe to have better treatment. According to Korea Biomedical Review, Korea recently…
Every game, the men of Northwestern Football leave the tunnel to a sign with an important reminder: “trust yourself.” Each player touches that sign before they head to the field,…
Every year, there are numerous unique ways to get involved in the rare disease community and help raise awareness. One example? The Harvest Hope for PWS event -- a virtual…
BRUKINSA (zanubrutinib), an orally administered Bruton's tyrosine kinase (BTK) inhibitor, has been approved in various countries for the treatment of mantle cell lymphoma (MCL), Waldenström's macroglobulinemia, and marginal zone lymphoma. For…
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