While there have been well-known racial disparities in the diagnosis and survival rates for breast cancer patients, there may be other factors besides the actual race of a patient causing…
A recent review, carried out by researchers from Spain and published in the Orphanet Journal of Rare Diseases (OJRD), discusses the rare condition alpha-1 antitrypsin deficiency. The original study, which…
The U.S. Food and Drug Administration (FDA) has just approved a treatment for smallpox. According to the World Health Organisation, there hasn’t been a known natural infection from smallpox since…
According to a story from currentargus.com, the annual meeting of the American Society for Clinical Oncology, which takes place in Chicago, is the largest convergence dedicated to cancer research in…
At 16 years old, I was told I would need a liver transplant within ten years. Being told at any age that you will eventually need a liver transplant is…
July is National Cleft & Craniofacial Awareness & Prevention Month! Cleft and craniofacial conditions affect thousands of infants, children, teens and adults in the US each year. Some are born with…
Last month at the United Leukodystrophy Foundation’s Family Conference, special education teacher Sharon Bergfeld, MASE, LBSI, LBSII, led a break out session on Student with Disability/Schooling Advocacy. The session provided…
According to Medical Xpress, a new study is underway that may change the future for those who have a condition called rheumatoid arthritis. Dr. Hazelwood from Cumming's School of Medicine…
A recent study, published here in the journal Nature Medicine and used as the source for this article, has found that certain kinds of cells may be useful for predicting…
An experimental gene therapy program (called AXO-AAV-OPMD) being developed for the treatment of oculopharyngeal muscular dystrophy has been licensed with exclusive global rights to Axovant Sciences from Benitec Biopharma. There…
Zogenix, Inc has announced the top-line results from a Phase 3 study of the investigational drug ZX008 (low-dose fenfluramine hydrochloride) for the treatment of young patients with Dravet syndrome. The…
In Matthew Hutson’s contribution to Medium’s “Future Human” series, he interviews George Church. Church, the Harvard geneticist, molecular engineer, and chemist has been hugely influential in the understanding of DNA.…
According to a story from PharmaTimes, the pulmonary arterial hypertension drug selexipag, developed by Actelion, was unable to make the cut to obtain coverage on the NHS in England. The…
Recently in Charcot‐Marie‐Tooth news, progress has been made in identifying better testing to use as outcome measures for people living with Charcot-Marie-Tooth disease type 1A. A study called “Outcome measures…
According to a story from Deseret News, after she was diagnosed with multiple myeloma, Jenny Ahlstrom almost immediately had to make crucial decisions regarding her treatment: decisions that could mean…
Zynerba Pharmaceuticals, Inc. has announced their plans to begin a clinical trial of Cannabidiol in children and teenagers who have Fragile X syndrome. The study, called CONNECT-FX, is expected to…
This is a continuation of An Interview with the Unstoppable Helena Baker. We had just learned about the multiple decades it took for her to receive her diagnosis of Fibula…
According to a July 9 report by the Muscular Dystrophy Association (MDA), the MDA awarded a large grant to researchers studying Charcot-Marie-Tooth disease. The grant, totaling over $1,000,000, was awarded…
The company Pharnext SA, which is developing the investigational drug PXT3003 for the treatment of Charcot-Marie-Tooth disease type 1A, has agreed upon a paediatric investigation plan for the drug with…
The Joshua Frase Foundation has released a new eighteen-minute documentary about the family’s fight for more research into gene therapies for myotubular myopathy, the rare condition that Joshua Frase was…
Danielle and Jonathan Foltz live on the New England coast with their three children. In an CNN article, Wayne Drash documents their meteoric fall from a mission family with big plans…
According to a June 20th report by the National Hemophila Foundation (NHF), the NHF along with several other prominent organizations in the bleeding disease community will be joining forces to…
According to a story from PBS, the FDA has been approving new treatments at a noticeably faster rate than it has in the past. As a result, it appears that…
BuzzFeed News reported a few weeks ago about the tragic story of 16-year-old Sara Manitoski who died of Toxic Shock Syndrome: a rare condition with a very common source. Toxic shock syndrome…
Happy Friday, everyone! We hope everyone's drinking water, wearing sunscreen, and taking a little time to relax this summer. As we get ready for the weekend, we've gathered four recent…
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