We seldom see articles about multiple system atrophy (MSA also called Shy-Drager syndrome). The disease is extremely rare and currently, there is no effective treatment. But according to a recent…
A person’s true feelings are often difficult to express during an interview. When asked a question by the interviewer there is no time to rehearse but only grasp at the…
Science 37 and Xperiome have entered into a partnership in order to allow for more viable rare disease research, according to an article in PR Newswire. If the partnership goes…
According to a recent article in the Seattle Cancer Care Alliance, the FDA’s recent approval of Axi-cel (Yescarta) will improve the chances of survival for relapsed or refractory follicular lymphoma.…
March is recognized each year as Bleeding Disorders Awareness Month, an event meant to help spread awareness about bleeding disorders among the general public and in the medical community. Bleeding…
First formed and launched within the UPstart incubator at the University of Pennsylvania's Penn Center for Innovation (PCI), biopharmaceutical company Linnaeus Therapeutics, Inc. ("Linnaeus") is now making strides in the…
On March 11, 2021, Patient Worthy attended an educational seminar hosted by the Cleveland Clinic titled Neuromyelitis Optica Spectrum Disorder (NMOSD): Educational Seminar for Patients, Family Members and Caregivers Focus…
Is it possible for certain gene mutations, which cause a specific condition, to also raise the risk of developing another condition? Well, says HealthDay, the answer might be yes. In a…
Molecular Targeting Technologies (MTTI), a radiopharmaceutical company, announced through Biospace that the FDA has given its approval for MTTI to move forward with a Phase 1 clinical trial to study…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
At first, everything in Amberlyn Smith's pregnancy seemed to be going smoothly. When she was just over 29 weeks pregnant, her screening tests appeared to be normal. However, at…
Patients with hereditary angioedema (HAE) experience recurrent "attacks," or periods of swelling under the skin. Now, in a Phase 2 clinical trial, researchers are evaluating PHVS416 for patients with…
Congress has recently introduced a new bill that would have a significant impact on the rare disease community if it passes. The legislation, called the Speeding Therapy Access Today (STAT)…
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,…
In a recent press release, biopharmaceutical company Hongkong Winhealth Pharma Group Co., Ltd. ("Winhealth") announced a new strategic cooperation agreement between itself and Merz Pharmaceuticals GmbH ("Merz"). Altogether, this agreement…
Past research has shown up to 83 genes and genetic loci associated with glaucoma. However, reports the Massachusetts Eye and Ear Infirmary in a news release, new research has identified…
It is estimated that one person in forty to sixty thousand is affected by Fabry disease. Approximately five hundred Canadians are known to have Fabry disease. Newswise recently carried the…
On March 11, 2021, global biopharmaceutical company Alkermes plc shared that its investigational drug candidate nemvaleukin alfa received Orphan Drug designation from the FDA. The treatment is designed for patients…
Altogether, there are over 7,000 rare diseases. Worldwide, 1 in 20 people live with a rare disease. But the journey to diagnosis can sometimes be confusing or difficult. So when…
Living with a rare disease can be difficult and scary for some, especially during a global pandemic that has affected millions. This Nottingham father knows these feelings firsthand; he lives…
According to a study from Hindawi, a team of scientists have reported the discovery of never before seen mutations that are linked to Alport syndrome, a rare kidney disease. These…
The month of March is recognized as Cerebral Palsy Awareness Month, a time that is dedicated to spreading awareness among the general public and the medical community about cerebral palsy,…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
In a recent press release, biotech company AltruBio Inc. shared that its therapy neihulizumab (AbGn-168H) received Fast Track designation for the treatment of patients with steroid refractory acute graft-versus-host disease…
Being diagnosed with a rare disease can bring on various emotions; everybody's experience is unique to them. Some feel relieved to finally put a name to their symptoms; others are…
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