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.
On March 7, 2024, the United Kingdom’s Medicines and Healthcare products Regulatory Agency shared via press release that the agency has approved Ztalmy (ganaxolone) for people living with CDKL5 deficiency…
Continue ReadingUK Approves Ztalmy (Ganaxolone) for CDKL5 Deficiency Disorder
Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
Prader-Willi syndrome (PWS) is a rare genetic disorder with a number of physical, emotional, mental, and behavioral manifestations. Individuals with PWS may exhibit cognitive impairment, hypotonia (poor muscle tone), and…
Continue ReadingPitolisant for Prader-Willi Syndrome (PWS) Earns Orphan Drug Designation
Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
Continue ReadingPGN-EDO51 Granted Orphan Drug Designation for Duchenne Muscular Dystrophy (DMD)
Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
Continue ReadingMarch 23: Lee University to Host the 2024 65 Roses 5K for Cystic Fibrosis (CF) Awareness
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.…
Continue ReadingRare Community Profiles: A Hidden Agony: Why James is Shedding Light on Geniculate Neuralgia and its Impact on Daily Life
The European Medicines Agency (EMA) explains that monepantel is an anthelmintic therapy, or a type of therapy that is used to expel worm-like parasites called helminths or nematodes from the gut.…
Continue ReadingPositive Study Results Available on Monepantel for ALS
An 18-year-old woman in Korea underwent a kidney transplant after developing end-stage kidney disease, for which she had been treated with dialysis. Doctors were unsure of the cause. While the…
Editor's Note: Chronic conditions and rare diseases don't discriminate, Patient Worthy and its partners are interested in amplifying the voices of those from all identities and backgrounds. If you have…
Continue ReadingShedding Light on Narcolepsy for Sleep Awareness Week 2024
Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which donor immune cells attack the recipient's tissues. The graft cells recognize…
Continue ReadingTopical Ruxolitinib Reduces Skin Lesions from Cutaneous GVHD
In February 2024, Jacob Bell reported in Biopharma Dive that pharmaceutical company Takeda Pharmaceuticals is planning on rapidly advancing late-stage testing of TAK-861 for people with type 1 narcolepsy (also…
Continue ReadingTAK-861: Takeda Plans to Begin Late-Stage Testing for Experimental Narcolepsy Treatment
In 2020, Chris and Susan Finazzo received news that would change the trajectory of their lives. They learned that their sons Chase (now 9) and Dylan (now 6) had a…
Continue ReadingMarch 9: CureDuchenne to Host “Champions in Miami” Event
In early March 2024, Clinical Trials Arena reports that global biopharmaceutical company Bristol Myers Squibb shared new data from the Phase 3 DAYBREAK study. Within the study, researchers explored how…
Continue ReadingNew Data Available from Phase 3 Trial on Zeposia for Multiple Sclerosis
In the rare disease community, fundraising plays a pivotal role in driving forward research and treatment initiatives, offering vital support to affected families, and guaranteeing access to necessary resources and…
Continue ReadingMarch 13: Team Matthew Luis Phelan-McDermid Syndrome Fundraiser
Corticosteroids are one of the standards-of-care for treating alopecia areata, alongside treatments such as topical sensitizers and Rogaine. However, none of these treatments have a high level of evidence in…
Continue ReadingSodium Valproate Shows Efficacy in Treating Patchy Alopecia Areata
Children with late-infantile onset Batten disease (CLN2) may be treated with an enzyme replacement therapy (ERT). However, this therapy cannot reverse the damage or progression children have already experienced. Researchers…
Continue ReadingExperimental Gene Therapy Showed Promising Safety and Efficacy in Primate Study of Late-Infantile Batten Disease
At the end of January 2024, global healthcare leader Eli Lilly and Company shared that its therapy OLUMIANT (baricitinib) received regulatory approval in Canada for the treatment of severe alopecia…
Continue ReadingICYMI: OLUMIANT Now Approved in Canada for Alopecia Areata
The 20th annual WORLDSymposium took place from February 4-9, 2024. During the course of the research conference, at least one presentation centered on data from the Phase 2 Mini-COMET long-term…
Continue ReadingWORLDSymposium: Nexviazyme Improves Ptosis in Infantile-Onset Pompe Disease
Having a chronic illness can be isolating. It can be difficult to share vulnerable and sensitive information about yourself with others, or to find ways to manage the trauma and…
Continue ReadingFewer than 33% of People with Alopecia Areata Attend Support Groups
The 17th Annual Congress of the European Association for Haemophilia and Allied Disorders took place this year from February 6-9, 2024. During the Congress, stakeholders came together to discuss how…
Continue ReadingEAHAD 2024: Positive Clinical Data on HMB-001 for Glanzmann Thrombasthenia
NY Rangers play hockey with little girl with Tyrosinemia Type 1.
When Jason Fox was just a child, doctors told his family that he would most likely not live to 18 years old. He had been diagnosed with Duchenne muscular dystrophy…
Imagine if you had recurring attacks of severe and unexplained swelling beneath your skin. For individuals living with hereditary angioedema (HAE), a rare inherited disorder, this isn't just a mere…
Continue ReadingExperimental Treatment Sebetralstat Shows Promise in Phase 3 HAE Study
Having a rare disease can be challenging and isolating. It's often difficult to describe the experience to someone who hasn't gone through it. Mobilizing funds for support, resources, and research…
Continue ReadingDrafting a Dream Team: Celebrating Breakthroughs from the February 2024 Uplifting Athletes Young Investigator Draft
Alzheimer’s disease is a progressive neurodegenerative disease and the most common form of dementia. People with Alzheimer’s disease may have difficulty remembering information, problems with learning, sleep disruptions, poor judgment,…
Continue ReadingCould Simulated Sunshine Improve Psycho-Behavioral Symptoms in Alzheimer’s Disease?
Zachary Thomas has been an advocate for the mucopolysaccharidosis type I (MPS I) community since he was born. Newborn screening is a public health initiative that tests newborn babies for…
Continue ReadingZachary Thomas Newborn Screening Act Could Introduce MPS I to Alabama’s Newborn Screening Panel
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