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 2015, the U.S. Food and Drug Administration (FDA) approved NUCALA (mepolizumab) for the treatment of severe eosinophilic asthma in adolescents and adults ages 12 and older, and by 2022,…
In mid-January 2023, Kristi Rosa of OncLive reported that the European Medicines Agency (EMA) granted Orphan Drug designation to pimicotinib (ABSK021) for inoperable tenosynovial giant cell tumors (TGCT). Orphan Drug…
Do you have cystic fibrosis? Are you searching for a stronger sense of community? Then BreatheCon, the unique virtual event spearheaded by the Cystic Fibrosis Foundation, is perfect for you! …
In the United States, companies must obtain approval from the U.S. Food and Drug Administration to run clinical studies and transport or distribute therapies across state lines. To do so,…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
In the past, Hyqvia [immune globulin infusion 10% [human] with recombinant human hyaluronidase] was approved to treat people with primary immunodeficiencies (PIs) ages two and older. Now, following data from…
In the European Union, Orphan drug designation is granted by the European Commission (EC) to drugs or biologics that are intended to diagnose, prevent, or treat rare conditions. The definition…
Both a hemithyroidectomy and total thyroidectomy are surgical interventions for people with low-risk differentiated thyroid cancer. Prior studies suggest that survival rates following these surgeries are relatively equal for these…
Unfortunately, clinical studies do not always work out the way they’re expected to. Biopharmaceutical company Mirum Pharmaceuticals, Inc. (“Mirum”) learned this after the Phase 2 EMBARK trial failed to meet…
On "The Golden Bachelor," 60-year-old Faith Martin started the season with a bang. She arrived on a motorcycle (badass!) before earning the First Impression rose, which my other Bachelor Nation…
A drug cannot be transported or distributed across state lines until its marketing application has been approved/cleared, so getting this clearance is crucial for many drug developers and physicians who…
In 2015, the United States approved Kanuma (sebelipase alfa) for the treatment of infants living with rare lysosomal acid lipase deficiency. However, the therapy was not approved in other countries;…
An estimated 90% of the global population is infected with Epstein-Barr virus (EBV). In some cases, this infection is asymptomatic; in others, EBV can lead to numerous health impacts, including…
Michael Strahan is well-known for being an American television personality, journalist, and former NFL Defensive End (DE) for the New York Giants. Now, the family man and proud father is…
In 2021, approximately 37 people per every 100,000 were diagnosed with idiopathic hypersomnia (IH), a neurological disorder characterized by excessive sleepiness and excessive daytime sleepiness. This amounts to roughly…
As it stands today, there are no cures for CDKL5 deficiency disorder. Treatment is symptomatic and supportive, and includes physical, occupational, and speech therapy. CDKL5 deficiency disorder causes seizures,…
AIV001 (axitinib): a potential, non-surgical therapeutic option for people with basal cell carcinoma (BCC). AiViva Biopharma describes AIV001 as: a novel formulation of a multi-kinase inhibitor combined with AiViva’s…
In Gibson City, IL, students at Gibson City-Melvin-Sibley High School (GCMS High School) are fighting to find a cure for Friedreich’s ataxia (FA). According to reporting from Jared Thomas…
The 66th American Society for Hematology (ASH) Annual Meeting and Exhibition took place from December 7-10, 2023. During the meeting, researchers and other stakeholders shared insights into clinical trends,…
In the United States, Breakthrough Therapy designation is granted by the U.S. Food and Drug Administration (FDA) to expedite drug development and review. This designation is granted to therapies that…
Healio Psoriatic Disease recently reported that Vyjuvek (beremagene geperpavec-svdt), a topical gene therapy designed for people with dystrophic epidermolysis bullosa (DEB), was granted Orphan Drug designation (ODD) by the Japanese…
In March 2022, the U.S. Food and Drug Administration (FDA) decided against the approval of oleogel-S10 for epidermolysis bullosa (EB); the FDA noted that, prior to approval, it would need…
In an investor news release from December 2023, Moderna and Merck shared follow-up data from the Phase 2b KEYNOTE-492/mRNA-4157-P201 clinical trial. During the trial, researchers explored the impact of Keytruda…
In December 2023, Merck and HiberCell announced that the organizations would be partnering on a Phase 1b clinical study to examine a drug combination for clear cell renal cell carcinoma…
Have you ever heard of Orphan Drug designation? This designation, granted by the U.S. Food and Drug Administration (FDA), is given to drugs or biologics that are designed to treat,…
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