Patient Worthy recently had the privilege to view a selection of short films about rare disease at Biotech Week Boston. These films highlight the stories of families and patients as…
On September 21, 2021, Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. The focus of this webinar was newborn screening in recognition of Newborn Screening Awareness Month, which is…
My daughter, Carson, was born in June 2020. I received a call 1.5 weeks later about Carson’s abnormal newborn screening. I was told it was most likely an error, but…
Rare Disease Week this year was July 14th-22nd and held virtually. With over 600 participates advocating for those living with a rare disease diagnosis and 250 patient organizations, this event’s…
Today’s report on compassion describes it as the resolve to serve others. This article, published by the Schwartz Healthcare Center in Cambridge, Massachusetts, features Victor Furtado, Security Director at NewBridge…
Cait Devin has always been entranced with music. This vocalist and guitarist, now 20 years old, released her first album in 2020, and is currently working on new music.…
An article recently appeared in the AAMC News celebrating the creation of The Children’s National Rare Disease Institute (CNRDI). The article described the long road patients with rare diseases must…
Rare Mother Meetup 6:00 PM, PST The Rare Mother Meetup will take place on the second Wednesday of each month and is hosted by Rare Mother Ashley Kenny. The meetup…
Jordana Rothschild, M.D. recently contributed an article to KevinMD discussing the perception that patients have about their doctors. Dr. Rothschild believes that the system we have created gives people the…
Clinical trial recruitment has always been challenging. It is especially challenging for therapies being tested for rare diseases, an already small population. Clinical trials for rare diseases are small out…
Guadalupe Hayes-Mota has worked for Biogen, Ultragenyx, Amgen, and GSK. He was the prior UCLA Health Director. Additionally, he is a member of the Massachusetts Rare Disease Advisory Council. He…
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…
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…
Rare Mother Meetup 6:00 PM, PST The Rare Mother Meetup will take place on the second Wednesday of each month and is hosted by Rare Mother Ashley Kenny. The meetup…
FoundationOne CDx is a comprehensive genomic profiling test that is tissue based. It is used to identify individuals diagnosed with ALK+ non-small-cell lung cancer (NSCLC) who will be eligible for…
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…
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…
What do phenylketonuria (PKU), galactosemia, and maple syrup urine disease (MSUD) have in common? All three are considered inborn errors of metabolism, or rare genetic disorders in which the…
Rare Mother Meetup 6:00 PM, PST The Rare Mother Meetup will take place on the second Wednesday of each month and is hosted by Rare Mother Ashley Kenny. The meetup…
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…
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