In November 2022, the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) approved Upstaza (eladocagene exuparvovec) for the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. The product, which…
In 1956, Dr. C. Miller Fisher first described Miller Fisher syndrome after seeing three patients who presented with absent tendon reflexes, ataxia (impaired coordination), and eye muscle weakness. Eventually,…
In the United States, the FDA grants Orphan Drug designation to drugs or biologics intended to diagnose, prevent, or treat rare conditions (those affecting fewer than 200,000 people in…
The 1980 World Series was a grand event for the Philadelphia Phillies. It was the last inning, and the Phillies were up by 3. With two players out and…
The Muscular Dystrophy Association (MDA) held its MDA Clinical & Scientific Conference from March 19-22, 2023 to share research, cutting-edge medical advancements, and clinical care practices within the muscular…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Dr. Amish Desai speaks with the utmost sincerity on KevinMD's recent podcast. The doctor explained the difficulty he encountered trying to balance treatment for his father while giving the same quality…
Albinism has not only been historically stigmatized, but has fueled superstition, misbeliefs, and discrimination in areas across the globe. For example, in certain areas, children with albinism may be…
A mother knows best. In Kaitlyn Fryar's case, her intuition about her son Hudson led her to pursue help from numerous doctors. According to CBS Pittsburgh, Hudson seemed to…
According to a story from MENAFN, the Delhi High Court recently issued a directive to the National Consortium for Research, Development, and Therapeutics for Rare Diseases to convene and move…
According to a story on msn.com, Alijah Cunningham is living with the rare disease Apert syndrome, resulting in the bones of his toes and fingers fusing together. Now three years…
Ezri Care Artificial Tears, as well as nine other ophthalmologic brands that create artificial tears or eyedrops, have been linked to dangerous Pseudomonas aeruginosa infections in people across sixteen different…
On March 9-10, 2023, Dynamic Global Events hosted the virtual Global Innovation in Patient Advocacy conference. This event was geared towards various stakeholders in the rare disease space and was…
An estimated 6,000-10,000 people within the United States are living with Rett syndrome. Prior to this month, these individuals have not had access to targeted treatment options. But this…
In many rare conditions, early diagnosis and treatment typically correspond with better outcomes. Prior to March 2023, LIVMARLI (maralixibat) was approved for the treatment of cholestatic pruritus related to Alagille…
On March 9, 2023, Issues Concerning Athletes held its 13th Annual Celebrity Bartender Night at Wasted Grain in Scottsdale, AZ. Erica Brooks, the Founder of Issues Concerning Athletes, spearheaded the…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
In many cases, babesiosis - a rare parasitic illness - is not fatal. Some individuals who are infected with babesiosis are even asymptomatic, meaning they don't show any symptoms.…
While there are existing therapies for individuals living with homozygous familial hypercholesterolemia (HoFH), a rare form of high LDL cholesterol, these therapies often fail to control cholesterol levels for…
CureDuchenne, the nation’s leading nonprofit dedicated to funding a cure for Duchenne muscular dystrophy (DMD) and a Patient Worthy partner, held its first “Napa in Newport” event in 2015;…
The Mississippi State University (MSU) Bulldogs joined forces with Children’s of Mississippi, the state’s only children’s hospital with statewide pediatric clinics, to honor pediatrics patients throughout Mississippi, sharing and uplifting…
The American Academy of Dermatology (AAD) held its Annual Meeting from March 17-21, 2023. During the meeting, stakeholders from the dermatology community connected to discuss clinical practices, research trends, and…
Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families,…
Jamas and Margot LaFreniere started the Sophie's Hope Foundation in 2020 shortly after Sophie, their daughter, was diagnosed with a rare disease: glycogen storage disease type 1B (GSD1B). The mission…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
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