Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.
21-year-old Chloe Feighery has a deep love and appreciation for her younger sister Heidi, who is just one and a half years old. When she learned that she was becoming…
Soon after Emma Young was born, she was diagnosed with a rare genetic disorder called Bannayan-Riley-Ruvalcaba syndrome (BRRS). At two months old, she battled COVID-19. Then she was diagnosed with…
For the first year of his life, Warner Kays looked like an average, healthy, happy baby. He crawled around, took joy in family time with his older sister Presley, and…
Have you ever heard of CD55-deficient protein-losing enteropathy? This rare condition, which is also called CHAPLE disease, was only identified in the past 10 years. Right now, there are only…
When Crystal York learned that her daughter Harmony had a rare genetic disease called DHX30, she was shocked. Even more shocking is that Harmony is just one of 40…
Chorea, a characteristic manifestation of Huntington's disease, refers to uncontrolled "dance-like" movements in the fingers, feet, face, or torso. These movements may worsen in times of heightened emotion or…
At the moment, there are limited treatment options for glioblastoma, a rare form of brain cancer. While surgery, radiation, chemotherapy, and electric-field therapy are all utilized in the fight…
The Phase 2 EXHALE-1 clinical trial, initiated by clinical-stage biotechnology company Areteia Therapeutics, Inc. ("Areteia"), sought to understand how safe, effective, and well-tolerated dexpramipexole was in reducing symptoms of…
For many people, a broken bone would severely interrupt their life. But six-year-old Avery Balcazar has learned to live with it—even when it’s tough. As reported by CBC News, Balcazar…
When Dr. Niranjana Parthasarathi, M.D., was in medical school at Virginia Commonwealth University (VCU), she still remembers a lesson that changed her life. Her course introduced her to systemic lupus…
A Phase 2 clinical study found TAK-994 to be relatively effective in reducing symptoms like excessive daytime sleepiness (EDS) and cataplexy (sudden, uncontrollable muscle weakness often triggered by strong…
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…
Despite advances in research and the advent of multiple investigational treatments, pancreatic cancer is still incredibly difficult to treat. The five-year survival rate sits at just 12%. Existing therapies…
Actress Francia Raisa is well-known for her roles in Grown-Ish, Bring It On: All Or Nothing, The Secret Life of the American Teenager, and How I Met Your Father. She’s…
When Camille Wahl was a child, she loved Irish step dance. She translated her love physically, becoming a competitive dancer until she was ten years old. It was then, reports…
Recent results are available from the dose escalation portion of the Phase 1 ANTLER study. In reporting from Targeted Oncology, Jordyn Sava shares that the study included 16 participants living…
In the United States, the FDA grants Fast Track designation to drugs that treat rare and serious conditions or fill unmet needs. The designation helps to facilitate and expedite the…
When Christine Todd learned that her kidneys were failing, she was frightened and concerned. Todd has polycystic kidney disease (PKD), an inherited condition characterized by cysts that develop in the…
In May 2023, the U.S. Food and Drug Administration (FDA) approved Vyjuvek, a gene therapy, for dystrophic epidermolysis bullosa (DEB). This treatment has brought support to numerous people living with…
As our understanding of genetics increases, so does our awareness of the multitude of genetic diseases—many of which continue to be discovered to this day. While X-linked lymphoproliferative disease (XLP)…
An estimated 25% of children globally are affected by atopic dermatitis; you may known this skin condition by its more colloquial name, "eczema." Often manifesting by five years old, atopic…
In the past, treatment for WHIM syndrome has focused on symptom management: treating infections and myelokathexis with antibiotics or granulocyte colony stimulating factor. Infusion can treat hypogammaglobulinemia and surgery…
If you’re looking for drive and determination, look no further. 14-year-old Sophia Nohre is ready to tackle any obstacle in her path—and she does so relentlessly. Her swimming coach, Adam…
In 2022, the U.S. Food and Drug Administration (FDA) approved DAXXIFY® (DaxibotulinumtoxinA-lanm) for the temporary treatment of fine lines and wrinkles. Many people touted DAXXIFY as a "Botox alternative."…
In June 2023, Michael, a 35-year-old man from Houston, started feeling ill and achy. By June 19, his symptoms had worsened significantly. Both Michael and his family believed that he…
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