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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In many areas of society, from healthcare to big business, companies are turning to artificial intelligence (AI) to solve pressing problems. Within the medical sphere, researchers recently developed an automated,…
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New AI Tool Can Evaluate Pediatric Hydrocephalus
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In early December, biotechnology company Aeglea BioTherapeutics, Inc. ("Aeglea") announced that its therapy ACN00177 received Rare Pediatric Disease designation from the FDA for the treatment of homocystinuria. Should this drug…
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ACN00177 Granted Rare Pediatric Disease Designation for Homocystinuria
In a recent press release, biopharmaceutical company Akari Therapeutics ("Akari") announced the publication of data on intravitreal nomacopan for patients with autoimmune uveitis. The data was sourced from a 2-year…
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New Data Published on Nomacopan Efficacy for Uveitis
Recently, the FDA expanded treatment indications for Gavreto (pralsetinib). According to MedPage Today, the drug was previously indicated for the treatment of patients with lung cancers with RET gene fusions. Now, this…
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Gavreto (pralsetinib) Now Indicated for RET-Mutant Thyroid Cancer
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In a press release from late November, pharmaceutical company BioCryst Pharmaceuticals, Inc. ("BioCryst") announced the publication of data from its APeX-J clinical trial in Allergy. During the trial, which was…
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Published Data Available from APeX-J Trial on Berotralstat for HAE
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Overall, Galapagos works to develop small-molecule treatments for patients with a variety of conditions. In a recent press release, the company reported positive top-line data from the Phase 2 PINTA…
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Positive Proof-of-Concept Data Reported on GLPG1205 for IPF
Obesity is considered to be excess fat accumulation that may lead to health issues. Worldwide, the prevalence of obesity has greatly increased over the years; the World Health Organization (WHO)…
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Imcivree Becomes First FDA-Approved Treatment for Genetics-Related Obesity
According to GuruFocus, the European Medicines Agency (EMA) recently validated a Marketing Authorization Application (MAA) submitted by biopharmaceutical company Mirum Pharmaceuticals, Inc. ("Mirum"). The MAA was centered around maralixibat, an…
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EMA Validates Maralixibat MAA for PFIC2
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On November 30, 2020, biopharmaceutical company Travere Therapeutics ("Travere") announced that patient enrollment was complete for the Phase 3 DUPLEX clinical trial. Within the trial, researchers will evaluate the safety,…
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All Patients Enrolled in Sparsentan Trial for FSGS
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According to Newswise, the FDA recently approved a new prostate cancer imaging technique developed by researchers at UCLA and UCSF. The imaging technique, called prostate-specific membrane antigen PET imaging (PSMA…
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FDA Approves PSMA PET Technique for Prostate Cancer from UCLA and UCSF
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Recently, biopharmaceutical company Acceleron Pharma Inc. ("Acceleron") announced that its investigational therapy ACE-1334 was granted Orphan Drug designation by the FDA. The treatment is designed for patients with systemic…
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ACE-1334 for SSc-ILD Granted Orphan Drug Designation
For the entirety of 2020, our lives have been dominated by COVID-19, the novel coronavirus linked to 64.5 million cases and 1.49 million deaths across the globe. In the United…
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A Possible Link Exists Between Endophthalmitis and COVID-19
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In 2019, Japan approved the use of EVRENZO (roxadustat) for patients with anemia of chronic kidney disease (CKD) who were also on dialysis. However, the orally administered therapy was not…
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Japan Approves EVRENZO for Anemia of CKD
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This past November, nearly 9,000 attendees showed up for the American Academy of Ophthalmology (AAO) 2020 Virtual Annual Meeting. During the meeting, biopharmaceutical company Kodiak Sciences Inc. ("Kodiak") announced that…
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Virtual Presentation Available on KSI-301 for wAMD, DME, RVO
When many people hear CAR T-cell therapy, they think about a treatment designed to address cancer. Recently, a new study done on mice models has suggested that an experimental form…
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Experimental CAR T-cell Therapy Explored for Retinoblastoma
According to HCP Live, University of Lübeck researchers recently connected rheumatoid arthritis (RA) and pemphigus, an autoimmune disorder characterized by blisters on the skin and mucous membranes. In fact, researchers…
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Having Pemphigus Increases RA Risk
Recently, scientists and researchers from the UCL Great Ormond Street Institute for Child Health (GOS ICH) and the UCL Cancer Institute worked to create an innovative solution for patients with cancerous…
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Novel CAR T-cell Therapy Shows Promise for Neuroblastoma
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On November 20, 2020, biopharmaceutical company RedHill Biopharma Ltd. announced the initiation of a Phase 3 clinical trial to study the orally administered RHB-204. The drug is designed to treat…
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Phase 3 RHB-204 Trial Begins for NTM Lung Disease
Recently, biotherapeutics company PureTech Health announced that their Phase 1 trial to evaluate LYT-100 (deupirfenidone) has completed. During the multiple ascending dose and food effect study, researchers analyzed the safety,…
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Phase 1 LYT-100 Study Shows Promise for IPF, Lymphedema
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Each year, thousands lose their vision to dominant optic atrophy (DOA), an inherited disease that negatively impacts the optic nerves. However, researchers and scientists from Trinity College Dublin may have…
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New DOA Gene Therapy Solution Developed
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When you think about genetic testing, what first comes to mind? Is it pancreatic cancer? While this might not be the first thing you consider, a new study suggests that…
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Genetic Testing Could Benefit Patients with Pancreatic Cancer
This past month, the Ice Bucket Challenge was brought back into the spotlight when co-founder of the ALS Ice Bucket Challenge, Pat Quinn, unfortunately passed away. As the ALS Association…
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Ice Bucket Challenge Funds Canadian ALS Guidelines
In a recent press release, biopharmaceutical company Akebia Therapeutics ("Akebia") announced the publication of the Phase 3 INNO2VATE study design and methodology. The publication examines the INNO2VATE trial, which evaluated…
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New Program Shows INNO2VATE Methodology: Vadadustat for CKD
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In a recent press release, the SynGAP Research Fund (SRF) shared that the Heller Laboratory of Neuroepigenetics would receive a $130,000 research grant. The fund, which will be granted over…
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SRF Awards Research Grant to Heller Lab of Neuroepigenetics
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In November 2020, Viking Therapeutics ("Viking") shared insight into their metabolic disease program. According to Greg Zante, the Senior VP (SVP) of Finance for Viking, the company's treatment VK2809, licensed…
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VK2809 for NASH Significantly Reduces LDL Cholesterol, Liver Fat