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.
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According to Medical XPress, researchers out of the Queen Mary University of London have been making strides in the field of medical research, particularly around pancreatic cancer. After identifying a…
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Novel Therapeutic Target Identified for Pancreatic Cancer
In early January 2021, biopharmaceutical company Avidity Biosciences ("Avidity") announced updates from its 2021 pipeline. Currently, Avidity is working to create a line of Antibody Oligonucleotide Conjugates (AOCs), therapeutic options…
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Avidity Announces 2021 Pipeline Updates For DM1, FSHD, DMD
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When breast cancer cells are biopsied, they are usually tested to determine if the cancer is hormone receptor-positive or hormone receptor-negative. Basically, if the cancer is hormone receptor-positive, estrogen and…
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Study Explores Androgens for ER+ Breast Cancer
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Oftentimes with cancers or rare conditions, there are subtypes of the cancer or disease. Breast cancer is no different. For example, a breast cancer diagnosis may encompass BRCA gene mutations or triple-negative…
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Did Scientists Find a New Treatment for Triple-Negative Breast Cancer?
On January 13, 2021, pharmaceutical company Marinus Pharmaceuticals, Inc. ("Marinus") shared via press release that the FDA gave the company a positive response following efficacy and safety data from the…
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Study Data Supports Filing Ganaxolone NDA for CDKL5
In a recent study, spearheaded by a team at the Postgraduate Institute of Medical Education and Research in Chandigarh, researchers determined that patients with non-cystic fibrosis (non-CF) bronchiectasis often had…
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A. fumigatus Infection Common in Non-CF Bronchiectasis
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Research has shown that there is a relationship between type 2 diabetes and cardiovascular disease, an umbrella of diseases that include damaged blood vessels, blood clots, and other issues. In…
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Finerenone for CKD May Reduce Cardiac Issues Associated with Type 2 Diabetes
If you have a rare condition and are also looking to start a family, you might question: how will my condition impact my pregnancy? Will I be able to have…
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Being Pregnant May Worsen Symptoms of CMT
Much of our news in 2020 was filled with headlines around COVID-19, the viral pandemic which now has over 96.2 million global diagnoses. As we enter into 2021, the numbers…
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Ultomiris Trial for COVID-19 Falls Short in Efficacy
Have you ever heard a song and heavily related to it? As though it was speaking directly to you? This is how I felt when I first heard German singer-songwriter…
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Singer with Benign Rolandic Epilepsy (BRE) Releases New Single
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…
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What are Genetic Counselors? An Interview with Dawn Laney, MS, CGC, CCRC
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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…
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IMBRUVICA Prescribing Information for WM Includes iNNOVATE Data
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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…
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Positive GENEr8-1 Trial Data Available for Hemophilia A
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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…
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United Therapeutics Sponsors PFF Registry
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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…
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Approvals Granted to Begin LYS-GM101 Clinical Trial for GM1 Gangliosidosis
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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…
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T. Gondii Infections May Increase Glioma Risk, Study Says
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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…
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DNMT3B Gene May Cause Pulmonary Hypertension, PAH Development
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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…
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BET Inhibitors Could Treat WM, Study Says
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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…
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Riabni Now FDA-Approved for GPA and MPA
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…
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DA01 IND Cleared for Parkinson’s Disease
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…
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Injecting Mosquitos with Human Malaria Advances Research
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…
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New Data Available for GSD1a, OTC Deficiency, and Wilson Disease Studies
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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…
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Generation Bio Overcomes Gene Therapy Limits with ceDNA in Hemophilia A
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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…
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BPK and Symbasis Sign MOU to Develop CLL Treatment
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…
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Patients to Be Recruited Into ASLAN004 Expansion Cohort for Atopic Dermatitis