The statistics are grim. A survey of 12,000 US nursing homes finds over 56,000 COVID related deaths as of July 2020. This number represents ten percent of all cases in…
Veer Basal, age 14, first started showing signs of Friedreich's ataxia when he was around ten years old. The first sign that something was wrong was when Veer began to…
In a recent press release, biopharmaceutical company Ultragenyx Pharmaceutical Inc. ("Ultragenyx") shared positive safety and efficacy data from Phase 1/2 clinical trials on gene therapy solutions for ornithine transcarbamylase…
In a press release from early January 2021, genetic medicines company Generation Bio Co. ("Generation Bio") announced data from a variety of studies. In one, the company shared that…
Recently, BiocurePharm, Korea ("BPK"), a subsidiary of biopharmaceutical company Brochure Technology Corp., shared that it had entered into a memorandum of understanding (MOU) with Symbasis GmbH ("Symbasis"). Together, the pair…
Kohl's has brought a new clothing line whose target audience is rare disease patients. The new clothing includes adaptive options to allow easy access to medical ports and easy dressing.…
Welcome to study of the week, a new series from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is…
BPD Bronchopulmonary dysplasia (BPD) is a rare condition that primarily affects the lungs of premature babies (though it can affect full term newborns as well). Most children with BPD are born…
In a recent press release, biopharmaceutical company ASLAN Pharmaceuticals ("ASLAN") announced that it would begin recruiting patients for an expansion cohort of a clinical trial evaluating ASLAN004 for patients…
According to a recent article in Statnews, providers must obtain prior authorization from an insurer for several reasons; to start, the insurer often requires physicians to justify prescribing a…
As reported in MSN, Duane Zingale explained that one aspect of being born with Treacher Collins syndrome is that you’re extremely memorable. Duane was born without ears or cheekbones. He…
Turkish citizens are putting pressure on their government in regards to access to rare disease treatments, specifically spinal muscular atrophy (SMA) therapies. There are currently two drugs available to Turkish…
Landon Groves was diagnosed with Kabuki syndrome, a rare multisystem disorder, and leukemia soon after his birth. Because of these conditions, his family faces hefty medical bills. His community has…
According to a story from dernieresactus.fr, the biopharmaceutical company Inventiva has recently announced plans for its upcoming phase III clinical trial. This trial will investigate its lead candidate lanifibranor as…
In early January 2021, pharmaceutical company Astellas Pharma Inc. ("Astellas") shared that its New Drug Application (NDA) and supplemental NDA for Myrbetriq (mirabegron) received Priority Review status from the…
Recently, clinical stage biotechnology company Prilenia Therapeutics B.V. ("Prilenia") announced that the first patient has been enrolled in a Phase 2/3 clinical trial evaluating pridopidine for patients with amyotrophic…
Note: This press release was republished with permission from the EveryLife Foundation for Rare Diseases. The Rare Disease Legislative Advocates (RDLA), a program of the EveryLife Foundation for Rare Diseases,…
Dr. Cory M. Resnick, Oral & Maxillofacial Surgeon at Boston Children’s Hospital and Assistant Professor at Harvard Medical School, describes the exciting progress he has made on the Prenatal Diagnosis of Pierre Robin Sequence. Interview by Philippe Pakter, chairman of Pierre Robin Europe.
Are you looking to get more involved in research, treatment, and awareness? Well, here's your chance! The University of Georgia (UGA) is currently seeking participants to join their study,…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
In some cases, combining different therapies can provide better overall outcomes for patients. However, in a Phase 2 clinical trial evaluating bevacizumab in conjunction with osimertinib for patients with EGFR-mutated…
One key struggle with managing rare diseases or other health conditions is finding the right treatment option. While bronchiectasis is usually treated with antibiotics and chest physiotherapy, there seems…
According to BioSpace, recent research has demonstrated that CRISPR gene editing could be the answer to treating progeria, or Hutchinson-Gilford progeria syndrome. The Broad Institute and NIH have collaborated to…
CBC News recently interviewed Amanda, Bradley, and Brad Bright, a close-knit black family living in Nova Scotia. The Brights explain that working their way through initial diagnosis and treatment…
My name is Megan and I live with acute hepatic porphyria (AHP). My health journey began with the arrival of puberty. I started to experience terrible nausea and abdominal pain…
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