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.
In October, I sat down with Laura and Ryan Nitahara to discuss their experience with Krabbe disease, a rare genetic lysosomal storage disorder and progressive neurological condition. Prompted by their…
In late December 2020, biopharmaceutical company AbbVie announced via press release that the prescribing information for IMBRUVICA (ibrutinib) is now updated to include long-term safety and efficacy data for…
On January 10, 2021, biotechnology company BioMarin Pharmaceutical Inc. ("BioMarin") shared positive data from its Phase 3 GENEr8-1 clinical trial. The clinical trial is evaluating an investigational gene therapy…
In a recent press release from mid-January 2020, the Pulmonary Fibrosis Foundation (PFF) announced that its registry, the PFF Registry, was being sponsored by biotechnology United Therapeutics. This sponsorship…
In an exciting press release, gene therapy platform company Lysogene announced that it had received approval from the Medicines and Healthcare products Regulatory Agency (MHRA) and Research Ethics Committee. As…
Did you know that a common food-borne illness might increase the risk of developing a type of rare brain cancer called glioma? According to Science Daily, a recent study suggests…
Recently, Pulmonary Hypertension News shared some burgeoning medical news: that the DNMT3B gene has now been linked to the development of pulmonary hypertension (PH) and a rare form of chronic high blood…
Waldenstrom macroglobulinemia (WM) is incredibly difficult to treat. In addition to having no cures, the condition only has one approved treatment option. However, researchers now believe that combining drugs, specifically…
According to ANCA Vasculitis News, the FDA recently approved Riabni (rituximab-arrx) for the treatment of adult patients with two specific subtypes of ANCA vasculitis: microscopic polyangiitis (MPA) and granulomatosis with…
In early January 2021, biopharmaceutical company BlueRock Therapeutics ("BlueRock"), in collaboration with the Memorial Sloan Kettering Cancer Center, announced via press release that its Investigational New Drug (IND) application for…
When you think of malaria, are mosquitos the first image that appears? While a microscopic parasite, Plasmodium falciparum, is responsible for malaria, mosquitos are often the vectors, carrying and transmitting…
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…
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…
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…
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,…
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 a recent press release from biopharmaceutical company Praxis Precision Medicines ("Praxis"), two of the company's drug candidates received either Orphan Drug or Rare Pediatric Disease designations for rare…
According to Parkinson's News Today, the first patient has been dosed in a Phase 1 clinical trial run by IMAC Holdings. The clinical trial is designed to evaluate mesenchymal…
In a recent press release, biotechnology company VISEN Pharmaceuticals ("VISEN") announced the approval of its Investigational New Drug application (IND) in China. The China Center for Drug Evaluation (CDE) accepted…
Looking for a feel-good story? Then look no further. In December, telecom company Cox worked to make a difference for the rare disease community. The organization headed its Elite Gamer…
For the last year, COVID-19 has dominated the headlines. The viral pandemic, present in over 200 countries and territories across the globe, is responsible for 86.7 million cases worldwide,…
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