In February 2024, Jacob Bell reported in Biopharma Dive that pharmaceutical company Takeda Pharmaceuticals is planning on rapidly advancing late-stage testing of TAK-861 for people with type 1 narcolepsy (also…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
Contributed by Jane Larkindale and Alayna Tress While millions of people globally are living with a rare disease, patients often find it difficult to feel seen or heard throughout their…
Contributed by Anna Ellis Every February 28, millions of people around the world participate in Rare Disease Day to raise awareness about the more than 10,000 identified rare diseases that affect…
Before reading, don't forget to head to Part 1 to learn more about Quris' founder Isaac Bentwich, why Quris was developed, and how the company is using its innovative bio-AI drug development…
Many people laud the accomplishments of clinical trials – and for, in some cases, good reason. Clinical trials have been crucial in identifying and developing therapeutic options for patients to…
Dr. Christopher Austin, a National Institute of Health Director for Transitional Sciences, spoke at last year’s virtual conference honoring Rare Disease Day. According to an article in the NIH Record, Dr.…
The Critical Path Institute (C-Path), formed in 2005, is a nonprofit, independent (public/private) partnership. The Institute evolved from the FDA’s 2004 White Paper known as the Critical Path Report and…
Collaboration can be extremely important when it comes to developing treatments, especially for rare disorders. Sharing information, technology, and expertise can lead to therapies that otherwise might not have seen…
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…
In early June 2021, SPG302, an investigational therapy for patients with amyotrophic lateral sclerosis (ALS), received Orphan Drug designation from the FDA. According to ALS News Today, the treatment…
According to a news release from April 2021, a new partnership is working to improve the commercialization of rare disease drugs for patients in China. Together, rare disease therapeutics company…
In a recent press release, biopharmaceutical company Aeterna Zentaris Inc. shared that the company would be developing a new therapy for patients with hypoparathyroidism. The preclinical development program will focus…
A report, commissioned by the National Organization for Rare Disorders and released by IQVIA, has revealed that the majority of orphan drugs, 79%, treat only rare diseases. Despite this fact,…
Raghav is a two-year-old boy born with an ultra-rare disease caused by a mutated GPX4 gene. He and eight other children are the only ones in the world who have…
In a recent press release, biopharmaceutical company Junshi Biosciences ("Junshi") announced a burgeoning collaboration with leading biologics platform company Coherus BioSciences, Inc. ("Coherus"). The pair is working to develop and…
In late January 2021, Dynacure, a clinical-stage drug development company focused on rare diseases, announced that it was granted a patent (U.S. Patent No. US 10, 865, 414) for its…
Before 1983, there were few treatments for rare diseases. Various obstacles stood in the way, including little representation for patients, few incentives, and a lack of resources. Fortunately, the Orphan…
Many medical professionals have applauded artificial intelligence and machine learning in the drug development process for a long time, as they can increase the speed while decreasing the cost. However,…
As reported in Science Daily, researchers at the University of Bristol’s MRC Epidemiology Unit working with pharmaceutical companies have developed an approach to reduce the failure rate of developing drugs.…
According to a publication from Specialty Pharma Times, the American Food and Drug Administration (FDA) has granted priority review to Tazemetostat, Epizyme, Inc's epithelioid sarcoma drug candidate. About Epithelioid Sarcoma…
According to a recent publication from BioPharma Dive, Dr. Janet Woodcock sat for an interview at the Biotechnology Innovation Organization's annual conference in Philadelphia earlier this month. Woodcock, the Director…
DrugPatentWatch recently published a copy of a paper originally published in the Journal of Applied Pharmaceutical Science. In the paper, authors Emanuel Almeida Moreira de Oliveira and Karen Luise Lang…
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