When Rhonda Stevey met her grandson, Koehyn, she was immediately smitten. The grandmother, who lives in Ohio, felt like her grandson was the happiest infant she had ever met. Unfortunately,…
Last week marked National Trigeminal Neuralgia Awareness Day, which was celebrated on October 7th. It's meant to raise awareness of this rare disease while providing support to those affected. You…
Written by Neil Smith I was labelled as a "late bloomer" as a teenager. I did not start puberty at the same time as my friends. Even when I started…
According to a recent article, the FDA has given approval for Apellis Pharmaceutical’s Inc.’s Empaveli to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). Paroxysmal Nocturnal Hemoglobinuria Paroxysmal nocturnal hemoglobinuria (PNH)…
Paul Williamson has always been a fiercely proud father and an advocate for the rare disease community. His son James was born with Smith-Magenis syndrome (SMS), a rare genetic developmental…
In a Case-Based Roundtable for Targeted Oncology, Dr. Haris Ali, an associate professor with the City of Hope Department of Hematology and Hematopoietic Stem Cell Transplantation, discussed a case of…
For many patients, symptoms of Huntington’s disease appear within midlife: between the 30s and 40s. This progressive disorder causes brain cell death, leading to a loss of physical and mental…
Fabry disease patients may have another treatment option soon, according to a press release published in BioSpace. Protalix Biotherapeutics and Chiesi Global Rare Diseases have provided an update on the…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, identify, or diagnose rare or life-threatening conditions. "Rare" conditions are those affecting under 200,000…
Data has recently been announced from a study investigating the efficacy of a new therapy called Alpelisib, for the treatment of PIK3CA-related overgrowth spectrum (PROS). The study was called EPIK-P1.…
Japan's Ministry of Health, Labour, and Welfare has just announced its approval of Alofisel, a stem cell therapy created by Takeda. This approval is specifically for patients who are diagnosed…
Acknowledgment: This story is sponsored by Otsuka America Pharmaceutical, Inc. and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing relevant,…
Acknowledgment: This story is sponsored by Otsuka America Pharmaceutical, Inc. and is promoted through the Patient Worthy Collaborative Content program. We only publish content that embodies our mission of providing…
To learn more about various conditions, how to treat them, and how to improve patient outcomes, medical research plays a crucial role. According to a news release from Saint Louis…
Although Jackson, age 62, has had CMT for ten years, he only divulged his illness this month. According to an article on eathis.com, Jackson chose a recently aired segment of…
Prior to October 8, 2021, there were no FDA-approved treatment options for pediatric patients with congenital athymia, an ultra-rare immune disorder. However, this all changed when the FDA approved Rethymic…
Patient Worthy makes a point to develop relationships with advocacy groups focused on rare diseases in order to help spread awareness, highlight patient experiences, and bring attention to community events.…
Did you know that just one tick bite could transmit to you a number of infections? That's right, according to a Lyme Science blog post from Dr. Daniel Cameron, MD,…
From October 7-11, 2021, the National Spasmodic Dysphonia Association (NSDA) is holding their 2nd annual Walk for Talk fundraiser to help raise awareness around, and funds for, spasmodic dysphonia, a chronic…
The first patient in a Phase 2a trial of BOS-580 as a treatment for nonalcoholic steatohepatitis (NASH) was recently dosed. Boston Pharmaceuticals, the developer of the drug, aims to address…
The 2021 Society for Hematological Oncology (SOHO) Annual Meeting took place last month from September 8-11, 2021. During the meeting, researchers discussed new advances in the field of hematology. According…
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…
With each new day and technological improvements come new discoveries about the world around us. Some of those discoveries center around medical conditions which have never been seen before. According…
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…
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