Have you ever had an itch that you just can't seem to scratch? Intensely itchy skin, also known as pruritus, is a hallmark characteristic of many conditions that affect the…
Did you know that June is considered Dravet Syndrome Awareness Month? Every year, people within the Dravet syndrome community come together to amplify stories, raise awareness, and encourage the general…
At the very top of the Rensselaer Polytechnic Institute's website reads a bold statement and call to action: "Why not change the world?" As one of the United States' most…
The World Orphan Drug Congress USA 2023 was held from May 23-25, 2023 at the Gaylord National Resort & Convention Center in National Harbor, MD. This conference is focused solely…
In late May 2023, clinical-stage biopharmaceutical company Sangamo Therapeutics, Inc. (“Sangamo”) shared that its product isaralgagene civaparvovc (ST-920) was granted Fast Track Designation by the FDA. This designation is…
A positive opinion is needed from the Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency (EMA), to begin marketing a drug. Once a positive…
WebMD recently carried a story about a young woman who, after nine years of wheelchair confinement caused by a rare metabolic disease with no name, was able to walk…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
As clinicians and scientists continue to delve into RNA sequencing benefits, they are learning how RNA sequencing provides insights for people with rare genetic conditions. The learning experience will provide…
On May 18, 2023, the Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. These webinars help provide updates to the rare disease community on legislation and other policy initiatives…
The energy in the crowd at American Family Field was electric. Everybody sat on the edge of their seat, waiting for the game to begin. If you’ve never been to…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
PhRMA Public Affairs recently carried the good news that Governor Holcomb of Indiana has joined the governors of Arkansas and West Virginia by signing a law aptly named 'Share the…
Last year, 35-year-old Jenna Neduchal ran her first Flying Pig Marathon. This annual event serves to unite the community, create an accessible event for all, and raise funds for important…
There are no cures or treatments available for people with hereditary spastic paraplegia (HSP) type 56, or SPG56. While the current standards-of-care focus on symptom relief and mobility assistance, patients…
On May 7, 2023, Kimberly Kotar completed a half-marathon. That’s impressive in its own right—only more so when you learn that, due to her transverse myelitis, Kotar was once unable…
In March 2023, 14-year-old Rylie Erbacher visited Washington, D.C. for a one-of-a-kind experience. The teen, who has spinal muscular atrophy (SMA) type 2, was one of just 10 people nationally…
Have you ever heard of the Orphan Drug designation? This designation, created through the Orphan Drug Act and granted by the U.S. Food and Drug Administration (FDA), is used to…
World Orphan Drug Congress USA 2023 May 23-25, 2023 The World Orphan Drug Congress brings together leading pharmaceutical and biotech companies, government and regulatory authorities, patient advocacy groups, payers, investors…
The National Organization for Rare Disorders (NORD), a Patient Worthy partner, formed in 1983 to provide support and education to people and families living with rare diseases, advocate for change…
On May 10, 2023, reports EMPR, the U.S. FDA approved Elfabrio (pegunigalsidase alfa-iwxj) for adults who are living with Fabry disease. Developed by Protalix Biotherapeutics in conjunction with Chiesi Global…
In 2014, head coach Jimbo Fisher led the Florida State Seminoles to win the BCS National Championship Game. He’s now head coach of the Texas A&M Aggies, recently deemed by…
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…
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