According to the Daily Sabah, the Health Ministry of Turkey is taking a new approach to family planning and screening for genetic disorders. As of October 8, 2021, the Health…
Most of us have heard of the fight-or-flight stress response. It becomes activated when we believe there is a chance we can outfight or outrun real or perceived danger and…
Dimeric calpains are calcium-activated cysteine proteases which help to regulate cell function. If these calpains are overactive, it could lead to fibrosis (tissue scarring) and tissue damage. In the…
Explaining something as complicated as a rare disease to a child can be difficult. That's why Alexion Pharmaceuticals created a books series titled "Inspired By." The series is a way…
Most patients with Norrie disease, a rare X-linked genetic disorder, often develop sensorineural hearing loss. But what mechanisms cause this hearing loss to occur? According to Medical XPress, researchers from…
A rare disease diagnosis can change your life; Mari Jackson knows this firsthand. She received a surprise diagnosis of pulmonary arterial hypertension (PAH) at age 48. Her symptoms began suddenly,…
At just 18 weeks old, Ivy Gaines, who was born prematurely, was diagnosed with spina bifida. His parents, Robert and Brittney, have worked tirelessly to ensure that Ivy is receiving…
On September 30, 2021, the National Organization for Rare Disorders (NORD) hosted an educational webinar titled "Rare Cancers: Breaking Down Barriers to Diagnosis, Treatment, and Research." The webinar focused on…
CurePSP has just announced their latest Venture Grant recipients. These grants total $320,000 and will all support the research of neurodegenerative disease treatments. CurePSP gives these grants twice every year.…
The Child Growth Foundation (CGF) is a leading UK charity which supports families, adults, and children with rare growth conditions such as SHOX deficiency, Silver Russell Syndrome, and Sotos syndrome.…
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…
Fenchol, a natural compound, is known for its herbal and appealing scent. This compound is found in a number of plants, including basil, nutmeg, and eucalyptus leaves. In the past,…
According to a recent press release from Horizon Therapeutics, UPLINZA, the only FDA-approved treatment for AQP4 antibody-positive neuromyelitis optica spectrum disorder (NMOSD), may be a viable treatment option for African…
Clinical trials face many obstacles regardless of which disease they aim to investigate. However, rare disease trials face additional problems that don't typically impact studies for more common conditions. Small…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
Emmalyn Reese Hudson was born 11 years ago at 35 weeks gestation weighing in at 5 lbs, 7 ozs. and 18 inches long. Our beautiful baby girl was so tiny…
"Rare" is a documentary that gives its viewers a glimpse into the world of rare diseases, something that many people know little about. Until now, the project has been funded…
To best address the needs of patients with rare diseases and rare cancers, additional treatment options are necessary. But developing these treatment options can be time-consuming and sometimes difficult.…
Screening tests are used to identify potential signs related to certain diseases or conditions. For example, newborn screening, performed at birth, gives insight into whether or not a child has…
Over the years, an inordinate amount of time and resources have been spent on the search for the cause of Alzheimer’s disease. According to a recent article in Medical News…
Ticks can carry a variety of infectious pathogens. Once the ticks bite humans, humans can then be infected with illnesses ranging from babesiosis and Powassan disease to Lyme disease. Now,…
Losing a loved one can be life-shattering. Ginny Toman knows this, as she lost her father to progressive supranuclear palsy (PSP) in 2019. Since then, she has learned how to…
If you could receive a vaccine to potentially prevent any condition, or at least reduce its severity, would you? According to Medical XPress, University of Toledo researchers are exploring this…
The EMA has recently published their recommendation for extending the indication of a treatment called Nucala to include patients diagnosed with relapsing-remitting or refractory eosinophilic granulomatosis with polyangiitis (EGPA). Specifically,…
Since 2012, the nonprofit organization Dysautonomia International has organized Dysautonomia Awareness Month. With the goal of spreading awareness of the condition in the medical community and among the general population,…
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