Patient Worthy was pleased to serve on the advisory committee for the development of this new resource. Caring for a child with a rare disease can be scary and confusing;…
The National Organization for Rare Disorders (NORD) has been a leading patient advocacy organization since the 1980s, helping advance the identification, treatment, and cure of rare disorders. In October 2021,…
Rare disease patients often face more obstacles and receive less aid than those impacted by more common conditions. There are many reasons for this inequity, but more research must be…
Developing therapies for rare diseases can be difficult, and it often requires effort from many different players. Recently, AllStripes has made large steps forward in rare disease research by securing…
During clinical trials, the FDA may sometimes order a clinical hold. This order is often given to halt or suspend trial operations if the FDA believes that participants would…
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…
When Caren was eighteen years old her parents were, in fact, given two options. If they were unwilling to consent to a lobotomy, Caren would remain in the dark ward…
The month of August is recognized as Stevens-Johnson Syndrome Awareness Month. The goal of this event is to elevate awareness about Stevens-Johnson syndrome among the general public and in the…
The month of August has been recognized as Autoinflammatory Awareness Month since 2015. This event is centered on spreading awareness about autoinflammatory illnesses among the medical community and the general…
Prior to this year, there were no reported cases of monkeypox in the United States since 2003. This all changed when a Texas man brought the rare disease back with…
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…
On July 21, 2021, Patient Worthy attended an online webinar presentation titled "How RDCA-DAP Can Help Inform Optimal Trial Design in Progressive Rare Disease." Organized by the Critical Path Institute…
The primary cause of death in melanoma patients is widespread metastases. Currently, there is no cure in cases of relapse or in advanced stages. A recent article in Science…
Have you ever heard of Bachmann-Bupp Syndrome (BABS)? If not, don't worry, you're not alone. In fact, BABS was only first discovered over the past few years. According to Medical…
The name of the organization is Disease InfoSearch. It is not only a resource for credible information but guides patients and families to valuable support groups and participation in…
This question is what the doctors at Massachusetts’ Cambridge Hospital were trying to determine. According to a recent article in the New York Times, a twenty-two-year-old man was rushed to…
Community support can be extremely uplifting and important, especially when one is impacted by a rare disease. This is the case for three-year-old Charlie Fry, who was born with SLC6a1.…
Dr. Jerry Vockley from the UPMC Children's Hospital of Pittsburgh has been studying the rare disease VLCAD for years. His work has led to the creation of two therapies which…
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…
According to an article in NCBI’s Drug In Context, it took the combined services of the internal medicine, neurology, and psychiatry departments to diagnose this rare disease. Anti-NMDAR encephalitis…
Accessing treatments can be difficult, especially if one is impacted by a rare disease. In fact, only 5% of rare diseases have an FDA-approved treatment. Because of this, many patients…
Researchers at NYU Abu Dhabi (NYUAD) have discovered the code that is associated with the liver’s genome (complete set of DNA) and its ability to regenerate. According to a…
As reported in PR NewsWire; a pivotal Phase II/III trial to create a therapy for activated PI3K delta syndrome (APDS) has announced they’ve successfully completed patient enrollment for a new…
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…
ANGEL AID CARES began as the brainchild of Cristol Barrett O’Loughlin, out of a shared desire to pay tribute to her own family experience and to uplift others in need.…
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