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.
In the National Football League's My Cause My Cleats campaign, NFL players are able to show their passions beyond the game and represent a cause that is important to them through customized…
It is extremely important to get the right medications into the hands of patients who need them. Recently, the FDA helped do this for pediatric patients (aged 2+) with primary…
Meniere's disease is a rare inner ear disorder characterized by "attacks" of tinnitus, ear pain, ear pressure, and dizziness. While doctors have long pondered the exact cause of this condition,…
In a recent news release, biopharmaceutical companies AbbVie, Inc. and SpringWorks Therapeutics, Inc. ("SpringWorks") shared that the two had entered into a joint clinical trial collaboration agreement. Through this collaboration,…
On December 10, 2021, biotechnology company Active Biotech shared that the first subject was dosed within a Phase 1 clinical trial evaluating laquinimod for non-infectious non-anterior uveitis. What is particularly…
In a recent news release, biopharmaceutical company Aligos Therapeutics, Inc. ("Aligos") shared that the first healthy volunteers were dosed in a Phase 1 clinical trial evaluating ALG-055009 for nonalcoholic steatohepatitis.…
On December 6, 2021, biotechnology company Longeveron Inc. ("Longeveron") announced that its investigational therapeutic candidate Lomecel-B earned Orphan Drug designation from the FDA. The therapy is designed to treat patients…
According to Pharmacy Times, the FDA recently granted Priority Review status to luspatercept-aamt (Reblozyl). The therapy, for which its developer has submitted a supplemental biologics license application (sBLA), is designed…
In 2016, Marley Berthold was born. But doctors, and her parents, noticed some concerning signs. Marley's silver hair had fallen out in clumps; she had macrocephaly (an abnormally large head)…
Nancy Yu co-founded AllStripes with a vision in mind: to unlock more treatment options for those with rare diseases. In Part 1 of our interview, Patient Worthy sat down with…
For as long as she can remember, Nancy Yu has been entranced by the intersection of science and technology. In fact, as she describes it, she imagines a world in…
How do cytotoxic T lymphocytes (TILs) play a role in ovarian cancer outcomes? What underlying biological activities or cellular interactions help stimulate TILs to destroy or damage cancerous cells? According…
In June 2021, Health Canada approved TRIKAFTA (elexacaftor/tezacaftor/ivacaftor) for patients with cystic fibrosis (CF), with at least one F508del mutation, aged 12+. According to a news release from late…
On December 2, 2021, Ultimovacs ASA ("Ultimovacs") announced via news release that its universal cancer vaccine, UV1, received Orphan Drug designation from the FDA. UV1 is designed for those…
In 2019, Connor Dobbyn, now 13, was diagnosed with Sanfilippo syndrome type C, also known as mucopolysaccharidosis III (MPS III). After his diagnosis, his parents came to recognize that there…
10-year-old Lyla McCarty has always dreamed of owning her own bakery. You see, Lyla has complex regional pain syndrome (CRPS) and often experiences periods of prolonged and intense pain. But…
According to a news release from the EveryLife Foundation for Rare Diseases, North Carolina became the 4th state in the nation to sign newborn screening legislation in 2021 (following Arizona,…
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. For the purpose of this status, "rare"…
According to Healio, the FDA recently granted Orphan Drug designation to nanatinostat and valganciclovir ("Nana-val") for the treatment of patients with Epstein Barr virus+ diffuse large B-cell lymphoma (DLBCL). In…
In April 2020, Elly Hawkins, a mother of three who currently lives in Queensland, Australia, began experiencing some odd symptoms. At first, the 35-year-old woke up with extreme vertigo. It…
In the first part of the "Save Our Medicine" series, you met moms Tiffany, DeAnna, and Sara, who are working to make a change and save experimental medication access for…
For Part 2 of "Save Our Medicine," we continue talking with Tiffany, DeAnna, and Sara about their families' NPC journeys, as well as the push for medication access. If you…
To learn more about Rachael's story, Niemann-Pick type C (NPC) symptoms, the diagnostic process, and how her mother Debbie found and offered support to other families, take a look at Parts…
According to a November 23 article from Muscular Dystrophy News, the FDA recently granted Orphan Drug designation to clinical-stage biotechnology company Regenxbio's experimental gene therapy candidate, RGX-202. The gene therapy,…
If your child was sick and there was a real, viable treatment option, the next step seems pretty straightforward: pursuing that option. But what if it wasn’t that easy? What…
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