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.
When asked his most important piece of advice for families within the rare disease community, Jeffrey Lord said: Do what you can control and move on from there. Every day,…
In his early stages of life, Austin (A.J.) Rose was diagnosed with an ultra-rare disease called pontocerebellar hypoplasia type 6 (PCH6). Now, at three years old, A.J. is a…
There seems to be no limits when it comes to what a parent will do for their child. And when it came down to it, David and Stacey Ogman knew…
In late September 2022, biotech company Oncoheroes Biosciences, Inc. ("Oncoheroes") shared via news release that its product dovitinib, which the company has an exclusive pediatric licensing agreement for from…
Over the last few years, there have been slight increases in the number of acute flaccid myelitis (AFM) cases throughout the United States. Specifically, 2014, 2016, and 2018 all saw…
In a late September 2022 news release from AVROBIO, Inc., the company’s gene therapy candidate AVR-RD-04 recently earned Rare Pediatric Disease designation. This investigational therapy is being developed as a…
According to an article in Batten Disease News, enrollment is complete for a Phase 1/2 clinical trial. Within the trial, researchers will be evaluating Batten-1 for adolescents and adults with…
Osteosarcoma is genomically complex, which means that – sometimes – it can also be difficult to treat. Treatment and management, however, are crucial to ensuring positive patient outcomes. Doctors and…
FBXL4-related mitochondrial disease urgently requires new therapies. Unfortunately, this condition is often fatal by early ages (early to mid-childhood). Therefore, it is important to spur research into potential interventions or…
Currently, an estimated 187,000 children within the United Kingdom, and millions worldwide, have some sort of arm disability. This can be linked to damage or various conditions, such as cerebral…
In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to prevent, diagnose, or treat rare conditions. These are conditions affecting fewer than 200,000 Americans.…
According to an article from Cancer Network, the U.S. Food and Drug Administration (FDA) recently granted Orphan Drug designation to second-line GNS561 (ezurpimtrostrat) for cholangiocarcinoma (bile duct cancer). In…
At 20 years old, Grace Hinchman was - for all intents and purposes - a happy and healthy young adult. Grace was studying Business at Loyola University Chicago and playing…
How does CAR T-cell therapy affect an enhanced anti-leukemia effect? Could higher doses influence toxicity or change patient outcomes? According to an article in the Pharmacy Times, researchers sought to…
Glioblastoma, a type of brain cancer, is notoriously difficult to treat. This is because the tumor is fast-moving, aggressive, invasive, and hard to remove with just surgery. However, an…
It has been four years since Amaria Granger, now age 14, was diagnosed with a rare disease called Niemann-Pick disease type C (NPC). Since being diagnosed with NPC, Amaria…
Did you know that September is National Sickle Cell Awareness Month? During this month, various stakeholders come together to share the stories of those affected by sickle cell disease,…
An unsettling fact: every three minutes, someone in the United States is diagnosed with some form of blood cancer. September is Blood Cancer Awareness Month to help raise awareness…
Before you read on, make sure you've checked out Part 1 of our interview, where we discussed what eosinophilic esophagitis is, its symptoms and diagnostic criteria, and why Dr. Dellon chose to…
The Orphan Drug Act of 1983 was designed to facilitate the development of orphan drugs, or drugs designed for patients with rare or life-threatening conditions. Now, the FDA grants…
Before you read on, please make sure to check out Part 1 of our interview, where we discuss what a desmoid tumor is, and share Amy's story of living with a desmoid…
The U.S. Food and Drug Administration (FDA) approved Dupixent for the treatment of moderate-to-severe atopic dermatitis in patients aged 6 months+; the drug is also approved as add-on maintenance therapy…
How is rheumatoid arthritis treated? Currently, the “gold standard” – referring to one of the most effective therapeutic methods – is a combination of methotrexate and adalimumab. This treatment is…
It is incredibly important to raise awareness of rare and underserved conditions; this increased awareness not only helps to spread the stories of those affected, but to garner support, connect…
Seven years ago, Dennis Smith started having extreme back pain. Dennis, 48 at the time, was extremely active. He loved playing football, going to the gym, and riding his bike.…
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