Rare Classroom: Anaplasmosis
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
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NICE Guidance Recommends Upstaza for AADC Deficiency
- March 31, 2023
Have You Heard of Miller Fisher Syndrome?
Zagociguat for Mitochondrial Diseases Earns Orphan Drug Designation
Did Toxic Turf Cause 6 Former Phillies Players to Develop Glioblastoma?
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PATIENT WORTHY SERIES
Rare Community Profiles: Diagnosed Before Birth: How the Sweatman Family Manages Their Son’s Hemophilia A
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,
Study of the Week: Scientists Develop More Effective Screening Method for Homocystinuria
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
Rare Classroom: Sturge-Weber Syndrome
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
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Duchenne Muscular Dystrophy
4-Year Proof-of-Concept Data Validates SRP-9001 for DMD
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
Anaplasmosis
Rare Classroom: Anaplasmosis
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
Idiopathic Pulmonary Fibrosis
A Doctor’s Struggle to Give Optimum Care to His Father and Patients
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
Albinism
NGO Condemns Media that Continues to Stigmatize Albinism
Albinism has not only been historically stigmatized, but has fueled superstition, misbeliefs, and discrimination in areas across the globe. For example, in certain areas,
TANGO2 disease
UPMC Launches TANGO2 Research to Help Families
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
Duchenne Muscular Dystrophy
Delhi High Court Calls for Duchenne Muscular Dystrophy Clinical Trial Funding
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
Apert Syndrome
Three-Year-Old Living with Apert Syndrome Needs Help to Afford Treatment
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
Rare Disease
Eyedrops Recalled After P. Aeruginosa Infections Killed 3, Injured 65
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
CDKL5
Global Innovation in Patient Advocacy: Navigating Clinical Trials for Rare Disease
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
Rett Syndrome
FDA Approves Daybue (Trofinetide) for Rett Syndrome
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
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