A Phase 3 study of upadacitinib, an ulcerative colitis treatment, met both its primary and secondary endpoints, according to an announcement from AbbVie. The trial, titled U-ACCOMPLISH, saw a third…
As the pandemic rages on, doctors across the country are beginning to notice a trend. A number of children who have contracted COVID-19 have also been impacted by a rare…
The NCCN, or National Comprehensive Cancer Network, has recently published treatment guidelines for children living with a rare kidney cancer, Wilms tumor. This is the organization's first guidelines written for…
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…
Tonix Pharmaceuticals and Inserm, which is the French National Institute of Health and Medical Research, have entered into a licensing agreement in which Tonix will hold the license to oxytocin-based…
Jason Kraft, the owner of Electric Bike Technologies, has brightened the lives of two sisters with two electronic tricycles that are typically made for senior citizens. Maggie and Charlie Monnin,…
Ocular Therapeutix has announced that they will attend and present at the Angiogenesis, Exudations, and Degeneration 2021 Meeting, which is being held virtually due to the pandemic. They intend to…
A survey created by Eurodis, a European rare disease advocacy alliance, has shown that those living with a rare disease have a worse healthcare experience than people with a chronic…
Tom Kean, the Senate Republican Majority Leader in New Jersey, is leading the charge to fully realize a recently partially approved bill, which would create a New Jersey Rare Disease…
Biotech company Ovid has released disappointing results from a trial of OV101, a potential treatment for Angelman's syndrome. The Phase 3 trial demonstrated that there was no significant difference in…
Recent research has discovered a new, potential treatment option that could provide an early intervention for Beckwith-Wiedmann syndrome and Russell-Silver syndrome. Published in Cell Reports, this study could provide the…
Genetic testing is being used more frequently to screen people for the hereditary risk of cancer, according to MedCity News. While advancements have been made and research is ongoing, there…
Alterity Therapeutics has recently announced the the Michael J. Fox Foundation will be funding a dose optimization trial for ATH434. According to BioSpace, this grant is the second from the…
Genethon and WhiteLab Genomics have entered into a partnership agreement, which will allow them to further develop their work in artificial intelligence and gene therapy. Within the terms of the…
Learning about rare diseases is one of the first steps towards advocacy. If you or a loved one are affected by cold agglutinin disease (CAD), you may want to get…
11-year-old Oliver Shultz is recovering at home after two weeks of stress and fear. It began with an ache in his back, followed by a fever. Soon, the family was…
According to BioSpace, Homology Medicines has released the first round of data from the clinical trials of HMI-203, a gene therapy being developed for the treatment of Hunter syndrome, also…
Aibe Shreve is only four years old, and he has already overcome so many obstacles in his short life. The little boy from Ohio Valley was born with two holes…
Vance Robinson was diagnosed with inclusion body myositis over ten years ago, and since then he has worked to raise money and awareness. Pre-pandemic, Vance would throw the first pitch…
Pfizer and Imcyse have entered into a collaboration and licensing agreement for a rheumatoid arthritis (RA) program that utilizes Imcyse's Imotope technology. The two companies have worked together in the…
Ionis and the University of California, San Diego (UCSD) have formed a team to investigate antisense oligonucleotide technology as a treatment for multiple myeloma. Their drug, ION251, has shown positive…
AVROBIO has released data from the ongoing Phase 2 FAB-GT trial, which is investigating AVR-RD-01 as a treatment for Fabry disease. This ex vivo lentiviral gene therapy was shown to…
The United Kingdom has taken a big step in the right direction for rare disease patients, as they have recently created the UK Rare Diseases Framework. This framework is intended…
An accumulation of alpha-synuclein is a major characteristic of Parkinson's disease, one that is often used to diagnose the condition. Current methods see medical professionals measuring the rate at which…
Zymeworks Inc. has recently began enrollment for an expansion cohort of the Phase 1 trial of ZW49. ZW49 is an antibody-drug conjugate (ADC) that targets HER2 positive cancer cells. This…
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