Parents of children who have received a diagnosis of Hutchinson-Gilford progeria syndrome (HGPS) may be relieved to learn the FDA recently approved Zokinvy capsules that were developed to lower…
The initial CRISPR treatment for sickle cell disease was administered to Victoria Gray in July 2019. Victoria, a resident of Forest, Mississippi, was born with sickle cell and has…
Research During COVID Many medical research projects have faced hurdles since the pandemic has started. Regulators have changed development timelines, and many have had to delay their clinical trials due…
The Partnership A new partnership has just been announced between Mendelian, a digital health company for rare disease, and Modality NHS partnership, a rare disease diagnosis program. Together, these organizations…
As reported in PR NewsWire; in an effort to help the entirety of the sickle cell disease community, Emmaus Life Sciences, Inc has announced a program that will provide their…
Eurordis The European Organization for Rare Diseases (Eurordis) is a nonprofit organization which organizes over 930 rare disease groups into one alliance. Groups have joined the network from 73 different countries.…
According to a story from empr.com, a patient living with Wilson's disease was recently awarded $28 million from a lawsuit after a jury found that the patient's doctor had failed…
Blueprint Medicines Corporation has just announced that they have submitted a supplemental New Drug Application (NDA) to the FDA. This application is for a therapy called AYVAKIT, which was developed…
Researchers have uncovered a new way to treat cancer that could lessen the side effects that patients often experience from chemotherapy. At the same time, it improves the curative power…
No deal Brexit means that the UK will be excluded from the 24 ERNs (European Reference Networks). Without the sharing of information, data, and research across countries, rare disease research…
Pulmonary fibrosis is a condition that is caused by scarring of the lungs which can lead to shortness of breath and other respiratory issues. Some cases of COVID-19 have lead to…
Many rare disease patients understand the financial burdens of living with a rare disease. As the therapies are only supporting a small number of patients, the price tag is often…
Complex Regional Pain Syndrome Complex regional pain syndrome (CRPS) is a rare condition that causes prolonged pain after a minor injury. This pain is severe and can be debilitating. One…
As the pandemic has raged on, we have learned more and more about what side effects can occur from severe COVID-19 infection. Some of these are common in patients with…
There is a large stigma against those with disabilities. Many misunderstand the wide diversity in disability and the success that those who are disabled can have in their lives. This…
by Lauren Taylor from In The Cloud Copy The Cardiometabolic Health Congress announced that they would be holding a virtual event due to the COVID pandemic that would cover various…
According to a story from Charcot-Marie-Tooth News, the National Organization for Rare Disorders (NORD) is beginning a new initiative that will be the focus of a public virtual meeting that…
by Lauren Taylor from In The Cloud Copy A cerebral venous thrombosis or CVT can happen when a clot is formed in the venous sinuses of the brain. This clot…
According to a story from ktla.com, a man from Nevada in his twenties has died after being hospitalized. The man was killed by hantavirus and was likely exposed to the…
According to a story from Globe Newswire, the biopharmaceutical company Ultragenyx Pharmaceutical Inc. has recently announced that its investigational treatment UX701 has earned the Orphan Drug designation from the US…
For those of us who suffer from depression, that mostly invisible mental illness that, at its worst, can render us incapable of getting out of bed and, at its best,…
On December 9th, 2020, a special edition of "Behind the Mystery: Rare and Genetic" will air on Lifetime TV as part of the acclaimed morning program The Balancing Act. It…
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…
The Orphan Drug Act originated in 1983 as an incentive for pharmaceutical companies who worked on developing treatments for rare disease. Since rare diseases are well, rare, choosing to work…
We all know that diagnosing cancer earlier means better outcomes for patients. It’s also essential to monitor the spread of cancer in already diagnosed patients. If we can watch as…
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