Caring is Sharing and Getting Involved. On April 17th Landmarks all over the world will be asked to ‘Light It Red’ in honor of Hemophilia Day. The theme of this…
According to a story from The Hill, the Centers for Disease Control and Prevention (CDC) has issued a new report which states that rates of tick-borne disease have increased 25%…
In late February/early March 2023, Corby Davidson had some news to share. Normally, as a longtime host on The Ticket—a sports radio station—Davidson’s discussions were rife with the latest…
Discovering a treatment for a rare disease is challenging but it can also be rewarding. This is especially true when treating an extremely rare disease such as perivascular epithelioid…
Ayla Bashir and her parents, Sobia Qureshi and Zahid Bashir, have participated in a medical first with Ayla surviving Pompe disease. The same disease caused the death of her…
According to a press release from the US Food and Drug Administration (FDA), on March 24, 2023, the agency issued a new draft guidance titled Clinical Trial Considerations to Support Accelerated…
STATHMIN-2 (STMN2) is a protein that has been proven to play a role in axonal development, repair, and stability. This protein is highly expressed in human motor neurons; research…
According to reporting for Cure Today, the FDA recently granted Accelerated Approval to a combination treatment of Padcev (enfortumab vedotin-ejfv) and Keytruda (pembrolizumab) for advanced or metastatic urothelial carcinoma.…
In the United States, Orphan Drug designation is granted by the U.S. Food and Drug Administration (FDA) to drugs or biologics that are being developed for rare conditions. Rare…
Medical research is incredibly important, especially within the rare disease community. Rare conditions tend to be under-researched and under-funded; this can make it difficult not only to better learn…
In the past, there has been limited research exploring the presentation of melanoma in Asian American and Pacific Islanders (AAPIs). Recent research has sought to explore this relationship and…
In 2022, the U.S. Food and Drug Administration (FDA) examined the potential of approving palovarotene, a selective RARγ agonist, for the treatment of fibrodysplasia ossificans progressiva (FOP). However, the FDA…
According to a story from LA Weekly, a lot of media coverage concerning cannabis has been largely positive in recent years. The medicinal value of CBD, for instance, has seen…
In November 2022, the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) approved Upstaza (eladocagene exuparvovec) for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. The product, which…
In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to diagnose, prevent, or treat rare conditions (those affecting fewer than 200,000 people in…
The 1980 World Series was a grand event for the Philadelphia Phillies. It was the last inning, and the Phillies were up by 3. With two players out and…
According to a story from MENAFN, the Delhi High Court recently issued a directive to the National Consortium for Research, Development, and Therapeutics for Rare Diseases to convene and move…
According to a story on msn.com, Alijah Cunningham is living with the rare disease Apert syndrome, resulting in the bones of his toes and fingers fusing together. Now three years…
On March 9-10, 2023, Dynamic Global Events hosted the virtual Global Innovation in Patient Advocacy conference. This event was geared towards various stakeholders in the rare disease space and was…
An estimated 6,000-10,000 people within the United States are living with Rett syndrome. Prior to this month, these individuals have not had access to targeted treatment options. But this…
CureDuchenne, the nation’s leading nonprofit dedicated to funding a cure for Duchenne muscular dystrophy (DMD) and a Patient Worthy partner, held its first “Napa in Newport” event in 2015;…
March 24 is being recognized this year as Lipoprotein(a) Awareness Day, a time to spread awareness among the general public and the medical field about elevated lipoprotein(a), a condition which…
Prior to February 2023, there were no FDA-approved treatment options for non-central nervous system manifestations of alpha-mannosidosis. But in mid-February 2023, Chiesi Global Rare Diseases shared via news release…
According to a story from people.com, beverage entrepreneur and former star on Real Housewives of New York City Bethenny Frankel has taken to the social media app Instagram to share…
Raising rare disease awareness is incredibly important - not just to spur research, but to validate the experiences of those within this community. Around Rare Disease Day on February…
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