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.
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…
When Adrienne Vollmer first learned that her son Graham had spinal muscular atrophy (SMA), she was shocked. At the time, there were no FDA-approved treatments (three now exist). The life…
At one point, Adam Isaac thought that he might become a professional golfer. But, sidelined from injury, he poured his heart and soul into music. His efforts played off;…
NS Pharma, Inc., a subsidiary of Nippon Shinyaku Co., recently announced via news release that its therapy NS-089/NCNP-02 was granted Orphan Drug designation from the U.S. Food and Drug…
Prior to launching a clinical trial, the FDA must clear an Investigational New Drug (IND) application. This allows the drug to be distributed across state lines, which is important…
Even with available therapies, pancreatic cancer is incredibly tough to treat and comes with a poor prognosis. Only 12% of people diagnosed with pancreatic cancer are still alive after…
In a late July 2023 news release, global healthcare company Grifols shared that positive topline data was now available from a Phase 4 clinical study evaluating XEMBIFY (immune globulin…
Between 2019 and 2020, the number of reported Hansen’s disease (leprosy) cases across the United States fell. However, there have been an uptick of cases since 2020, especially in…
For as long as she can remember, eighth-grader Ella Bork has held a keen knowledge of rare diseases, especially Alagille syndrome. Her mother, Cher, is actually the Executive Director of…
65 roses. This phrase is often used in reference to cystic fibrosis; in fact, the origin of 65 roses is a young boy who couldn’t properly pronounce the name…
Each year, there are less than 600 new cases of papillary craniopharyngioma (PCP) diagnosed within the United States. While PCP typically has a good prognosis with treatment, treatment is…
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