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.
According to a news release, researchers from biopharmaceutical company HUTCHMED and pharmaceutical company AstraZeneca launched, at the onset of November 2021, the Phase 3 SAMETA clinical trial. During the trial,…
In Part 1 of Rachael's story, I spoke with her mother Debbie about what Niemann-Pick type C (NPC) is, the beginning of the diagnostic journey, and how Dr. Behar helped…
Since the onset of COVID-19, there have been approximately 262M cases, and 5.2M deaths, on a worldwide scale. However, the introduction of various COVID-19 vaccines, such as those from Pfizer…
In Part 1 of "The TSC Navigator: A New Resource to Guide Families through the TSC Journey," I sat down with Ashley Pounders to discuss the TSC Navigator, its purpose,…
“Hi, I’m Rachael, and I’m forever 33.” When Debbie Kaflowitz remembers her daughter, she thinks about everything that Rachael loved to do. Rachael enjoyed dancing and performing in ballet recitals,…
In mid-November 2021, global biopharmaceutical company Zai Lab Limited ("Zai Lab") shared via news release that the first patient was dosed in the Greater China portion of the ADHERE study.…
The TSC Alliance® first began as a grassroots organization, founded by motivated mothers whose children had been diagnosed with tuberous sclerosis complex (TSC), a rare genetic disease. Since its founding,…
Nancy Green and Judi Beckerleg first met nearly 22 years ago in 1999. After Judi moved to Arizona, she began working as an aide in Nancy's classroom, where Nancy taught…
According to a late October 2021 news release from biopharmaceutical company Mereo BioPharma Group plc ("Mereo"), the company's therapeutic candidate alvelestat received Orphan Drug designation from the FDA. The therapy…
A Tyler, Tx artist, mother, wife, and friend has her community rallying behind her in her fight against oligodendroglioma, a primary central nervous system cancer. According to the Tyler Morning…
Orphan Drug designation in the United States is granted to drugs or biologics intended to treat, prevent, or diagnose rare diseases or conditions, defined as those affecting under 200,000 Americans.…
Currently, there are no therapeutic options available for patients with myotonic dystrophy type 1 (DM1). However, that may soon change. According to Marketwatch, the FDA recently granted Fast Track…
In 2019, the United States approved Vumerity (diroximel fumarate) to treat adult patients with relapsing-remitting (R/R) multiple sclerosis (MS). Now, two years later, the European Union has approved this…
It is always exciting to see the onset of a new clinical trial designed to research therapies to fill unmet needs for patients. According to a news release from biotechnology…
In the sports world, former Atlanta Hawks president Bob Williams has been known for his tenacity - but according to Newsweek, he may soon be known for something different: contracting…
On October 18, 2021, biopharmaceutical company Paratek Pharmaceuticals, Inc. ("Paratek") shared via news release that the first patient had enrolled in a Phase 2b clinical trial. Within the trial, researchers…
In a recent news release from global specialty pharmaceutical company Kyowa Kirin International PLC ("Kyowa Kirin"), the company announced the release of new real-world data on POTELIGEO (mogamulizumab) for patients…
Clinical trials are fantastic ways to develop a deeper understanding of a specific disease, as well as determine the safety, efficacy, and tolerability of potential treatments. Unfortunately, these treatments are…
When she was just nine years old, Maika Ting, from California, was diagnosed with aplastic anemia, a rare and serious blood disorder. Although her brother was a bone marrow match,…
Years ago, talabostat (BXCL701) failed during a Phase 3 clinical trial to evaluate the therapy for patients with pancreatic cancer. However, drug developer BioXcel Therapeutics (“BioXcel”) did not give up…
I've certainly never been a runner -- and neither was Diane Owens, at least for a while. The mother, who lives in Southbury, CT, admits that it used to be…
In the past, many doctors and researchers have considered malaria, a parasitic mosquito-borne illness, to be an infection of the blood and liver. As malaria can cause anemia (low red blood…
3-year-old Ruby Ann Grimes is bubbly, happy, and loving. But each and every day, Ruby faces an ongoing battle: Opsoclonus-Myoclonus syndrome (OMS). Last January (2020), Ruby was first diagnosed with…
According to a November 3, 2021 news release from biopharmaceutical company OS Therapies, the company’s therapeutic candidate OST-HER2 (OST31-164 / Listeria monocytogenes) received Rare Pediatric Disease Designation from the FDA.…
In late October 2021, the National Institute for Care and Excellence (NICE) in England recommended Givlaari (givosiran) to treat patients with acute porphyria -- or, more specifically, to treat patients…
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