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.
Over the last few months, researchers have been enrolling and dosing patients with hereditary angioedema (HAE) from Europe, Israel, and Canada within the Phase 2 RAPIDe-1 clinical trial. According to…
In case you missed it - a news release from biopharmaceutical company Pharnext SA ("Pharnext") shared that on July 12, 2021, the first European patient enrolled in the Phase 3…
Sometimes, different surgical techniques are required to operate on adults as opposed to children. For example, surgical revascularization is used to treat pediatric patients with Moyamoya disease - though it…
In the past, ibrutinib has been used to treat patients with a variety of conditions, including chronic lymphocytic leukemia (CLL), mantle cell lymphoma (MCL), and chronic graft-versus-host disease (cGVHD). According…
In mid-July 2021, biotechnology company Alterity Therapeutics ("Alterity") shared the publication of new data regarding ATH434 for patients with multiple system atrophy (MSA). The data comes from a preclinical…
In a recent news release, rare disease-focused biopharmaceutical company CANbridge Pharmaceuticals Inc. ("CANbridge") shared that the National Medical Products Administration (NMPA) of China accepted an Investigational New Drug (IND) application…
On August 2, 2021, biotechnology company Amicus Therapeutics ("Amicus") shared that its oral treatment Galafold (migalastat) was approved by the European Commission (EC) for the treatment of adolescent patients (ages…
According to a press release from late July 2021, LX1004, a treatment for patients with CLN2 Batten disease, received both Orphan Drug and Rare Pediatric Disease designations from the…
Clinical guidelines are often used to improve patient treatment. These guidelines use evidence reviews, as well as an examination of other standards-of-care, to optimize patient care and outcomes. According…
In the past, researchers have questioned whether an association exists between Lyme disease and psychiatric or mood disorders such as depression, anxiety, or even suicidal ideation. While some recognized a…
As of today, the world has been battling the COVID-19 pandemic for over 1.5 years. Throughout the world, the novel coronavirus is responsible for 197 million cases and approximately 4.7…
Despite drug developers' best efforts, the path to approval is not always easy. Ask biotechnology company Ardelyx, whose potential treatment option tenapanor was recently rejected by the FDA. According to…
If I told you that tiny bubbles could be used to more effectively treat cancer, would you believe me? Well, in the case of neuroblastoma, that might be correct. According…
On July 28, 2021, biopharmaceutical company Thetis Pharmaceuticals ("Thetis") shared that its small molecule Resolvin E1 (RvE1) therapy, TP-317, received Orphan Drug designation from the FDA. While TP-317 has…
In the United States alone, an estimated 300,000 people have moderate-to-severe chronic idiopathic urticaria (CIU), or chronic hives which are either recurrent or last for over 6 weeks, which…
Orphan Drug designation is a status granted by the FDA to drugs or biologics intended to treat patients with rare conditions. In this case, the FDA defines "rare" as…
Previously, biopharmaceutical company XyloCor Therapeutics ("XyloCor") performed the first portion (dose-escalation) of the Phase 1/2 EXACT clinical trial evaluating its investigational treatment XC001 (encoberminogene rezmadenovec) for patients with refractory…
According to a relatively recent news release from biopharmaceutical company Dicerna Pharmaceuticals, Inc. ("Dicerna"), interim data is now available from a Phase 1 clinical trial evaluating belcesiran for patients with…
According to a July 27 news release from biopharmaceutical company Day One Biopharmaceuticals ("Day One"), the company's therapeutic option DAY101 received Rare Pediatric Disease designation within the United States. Altogether,…
In a news release from July 21, 2021, biotechnology company Genentech shared that its therapy Venclexta (venetoclax), in conjunction with azacitidine, received Breakthrough Therapy designation from the FDA. Overall, the…
Sometimes, clinical trials do not go the exact way that is expected. According to Targeted Oncology, this is what happened in a Phase 1b clinical trial evaluating RVU120 for…
Unfortunately, there are a great deal of inequities within the healthcare system. Health inequity occurs when groups are treated differently, offered different or worse care, or have a lower…
According to a press release from July 15, 2021, the Chinese NMPA recently approved an Investigational New Drug (IND) application submitted for CAN106, a potential therapeutic option for patients with…
Near the end of June 2021, pharmaceutical company Crinetics Pharmaceuticals, Inc. ("Crinetics") shared via news release that the first patient had been dosed in the Phase 3 PATHFNDR-1 clinical trial.…
Medical studies are crucial players in the journey to develop new and more efficacious treatment options for patients with rare diseases. On June 10, 2021, Vertex Pharmaceuticals Incorporated ("Vertex") shared…
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