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.
Internationally, over 400,000 people die of malaria each year. In many cases, a majority of these deaths occur in sub-Saharan Africa; children are the most vulnerable. However, according to Medical…
Continue ReadingNovel mRNA Vaccine Prevents Malaria in Mice Models
For the first time, a research study has discovered a genetic marker related to high-risk neuroblastoma. According to Medical XPress, researchers determined that anaplastic lymphoma kinase (ALK) gene mutations and…
Continue ReadingStudy Finds Genetic Marker for High-Risk Neuroblastoma
As reported by Oncology Nursing News, gunagratinib (ICP-192) was recently granted Orphan Drug designation by the FDA. Altogether, this treatment is being evaluated as a potential treatment for patients…
Continue ReadingICP-192 for Cholangiocarcinoma Granted Orphan Drug Status
Currently, there are no real treatments for patients with primary mitochondrial myopathy (PMM). Most therapies, such as physical, occupational, or speech therapy, are designed to simply lessen or mitigate symptoms.…
Continue ReadingICYMI: REN001 Receives Fast Track Designation for PMM
In late May 2021, the FDA granted accelerated approval to Truseltiq (infigratinib). As reported by Cure, this therapy is designed for adult patients with unresectable locally advanced, previously treated, or…
Continue ReadingTruseltiq Approved for Metastatic Cholangiocarcinoma
When Stephanie Ernst-Milner first discovered that she was having twins, she was speechless. But what she learned over the course of her pregnancy, and in the seven years since, has…
Continue ReadingRedefining TAPS: How Stephanie and the TAPS Support Foundation Raise Awareness
Mobility issues are common in patients with osteogenesis imperfecta (OI) - especially tendinopathy, or tendonitis. But why does this happen? According to Medical XPress, a study performed by Baylor College…
Continue ReadingFkpb10 Deletion Causes Tendinopathy in Osteogenesis Imperfecta
For years, researchers have been evaluating pridopidine, developed by Prilenia Therapeutics ("Prilenia"), as a potential therapy for patients with neurodegenerative conditions. As reported in HD Buzz, new research has highlighted…
Although it is still considered an experimental technique, gene therapy shows promise for treating a wide variety of genetic diseases or disorders. In a recent news release from the Sanford…
Over the past few years, Elmiron (pentosan polysulfate sodium, "PPS"), a therapy designed to treat patients with interstitial cystitis (IC), has been associated with potential vision damage. Despite a variety…
From June 5-8, 2021, the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) held its 58th Annual Virtual Meeting. Due to COVID-19, the meeting was held fully online. However, that…
Continue ReadingDapagliflozin Benefits Patients with FSGS, Study Shows
Some conditions or diseases are more prevalent in certain groups. In the case of idiopathic intracranial hypertension (IIH), new research suggests that it is much more common in Black or…
Continue ReadingIIH Rates are Significantly Higher for Black and Hispanic Women
Normally, cerebrospinal fluid (CSF) surrounds our brain and spinal cord, providing numerous benefits from injury protection and nutrient delivery to waste removal. Hydrocephalus, or "water on the brain," occurs when…
In a news release from late May 2021, biopharmaceutical company Timber Pharmaceuticals, Inc. ("Timber") shared that all patients have been enrolled in the Phase 2b CONTROL trial. During the trial,…
Continue ReadingAll Patients Enrolled in TMB-001 Trial for Congenital Ichthyosis
Could a specialized cancer vaccine actually improve patient outcomes? According to a news release from biotechnology company AIVITA Biomedical, Inc. ("AIVITA"), the answer could be "yes." Citing data from a…
Rare Pediatric Disease designation is a status granted by the FDA to drugs or biologics intended to treat rare diseases (affecting under 200,000 Americans) in patients aged 18 or younger.…
Continue ReadingJSP191 for SCID Granted Rare Pediatric Disease Designation
Typically, idiopathic intracranial hypertension (IIH) is associated with obesity, or a body mass index (BMI) over 30.0. As obesity rates have continued to increase globally, so have the incidences of…
Previously, Ocaliva (obeticholic acid) was approved as a prescription treatment for patients with primary biliary cholangitis (PBC), a rare and chronic liver disease. However, Market Screener reports that biopharmaceutical company…
Continue ReadingOcaliva Contraindicated for PBC with Decompensated Cirrhosis
At just four years old, Gabriel Spiro is facing his 3rd open-heart surgery. You see, Gabriel was born with hypoplastic left heart syndrome (HLHS), a rare and severe congenital heart…
Continue ReadingFamily Needs Help After $10k for Boy’s HLHS Surgery Stolen
Prior to 2021, there were no FDA-approved treatments for patients with type 1 plasminogen deficiency, or hypoplasminogenemia. As a result, there was an urgent need to fill this unmet need…
In late May 2021, biopharmaceutical company PTC Therapeutics, Inc. ("PTC") shared that its therapy, PTC923, received Orphan Drug designation within both the United States and Europe. The treatment is designed…
Continue ReadingPTC923 for Hyperphenylalaninemia Granted Orphan Drug Status
In a press release from late May 2021, biotechnology company CancerVAX, Inc. ("CancerVAX"), which works to develop targeted immunotherapy treatments, shared that it had entered into a sponsored research agreement…
Continue ReadingResearch Program Formed to Develop Ewing Sarcoma Immunotherapy
From June 5-8, 2021, the European Renal Association - European Dialysis and Transplant Association (ERA-EDTA) held its 58th Annual Congress. Though the event was held virtually, the meeting still expanded…
Continue ReadingIptacopan for IgAN Meets Phase 2 Trial Primary Endpoint
According to a June 11 press release from biotechnology company Equillium, Inc. ("Equillium"), positive topline data is now available from the Phase 1b EQUATE clinical trial. Within the clinical trial,…
Continue ReadingPositive Topline Data Available on Itolizumab for GvHD
Currently, the European Hematology Association (EHA) Virtual Congress 2021 is going on from June 9 through 17, 2021. During the Congress, hematologists and other stakeholders will discuss clinical research and…
Continue ReadingNew Safety and Tolerability Data Available on CAEL-101 for AL Amyloidosis
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