For the first year of his life, Warner Kays looked like an average, healthy, happy baby. He crawled around, took joy in family time with his older sister Presley, and…
Have you ever heard of CD55-deficient protein-losing enteropathy? This rare condition, which is also called CHAPLE disease, was only identified in the past 10 years. Right now, there are only…
A recent press release from Verrica Pharmaceuticals and its partner Lytix Biopharma reported in Healio announces the results of the first portion of a study evaluating VP-315 to treat basal…
When Crystal York learned that her daughter Harmony had a rare genetic disease called DHX30, she was shocked. Even more shocking is that Harmony is just one of 40…
The life of the four-year-old was saved. The operation conducted at Medcare Orthopedics was a success. It was the first of its kind to be performed in the UAE.…
Enterome presented its findings to the 17th International Conference on Malignant Lymphoma (ICML) in June of this year. As reported in the GlobeNewswire, the company demonstrated that E02463 together…
The later part of 2023 will see decisions on drugs in four areas such as advanced melanoma, sickle cell disease, Alzheimer’s disease, and depression. BioSpace shines the spotlight on…
Chorea, a characteristic manifestation of Huntington's disease, refers to uncontrolled "dance-like" movements in the fingers, feet, face, or torso. These movements may worsen in times of heightened emotion or…
At the moment, there are limited treatment options for glioblastoma, a rare form of brain cancer. While surgery, radiation, chemotherapy, and electric-field therapy are all utilized in the fight…
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…
The Phase 2 EXHALE-1 clinical trial, initiated by clinical-stage biotechnology company Areteia Therapeutics, Inc. ("Areteia"), sought to understand how safe, effective, and well-tolerated dexpramipexole was in reducing symptoms of…
My name is Nicholas Alves, and I am a 26-year-old male from Massachusetts. I have a completely life destroying condition called PSSD, or post SSRI sexual dysfunction. PSSD can arise…
This press release was provided by the Sudden Arrhythmia Death Syndrome Foundation (SADS), a Patient Worthy partner organization. Key facts: ● Externally-led patient-focused drug development meeting (EL-PFDD) with FDA hosted…
For many people, a broken bone would severely interrupt their life. But six-year-old Avery Balcazar has learned to live with it—even when it’s tough. As reported by CBC News, Balcazar…
When Dr. Niranjana Parthasarathi, M.D., was in medical school at Virginia Commonwealth University (VCU), she still remembers a lesson that changed her life. Her course introduced her to systemic lupus…
It was the second time 26-year-old Bruce Campbell had to deal with paralysis. At the age of six, he was paralyzed by Guillian-Barre syndrome which caused an attack on…
Sounds like ‘food for thought’. The term ‘sandwich generation’ describes family members who are usually between the ages of 45 and 55 and are responsible for raising their children…
A Phase 2 clinical study found TAK-994 to be relatively effective in reducing symptoms like excessive daytime sleepiness (EDS) and cataplexy (sudden, uncontrollable muscle weakness often triggered by strong…
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…
Authored by Dr. Harsha Rajasimha, Founder and Executive Chairman, IndoUSrare. Co-Authored by Dr. Padma Rammoorthy, Medical Consultant, IndoUSrare. The diagnostic journey for patients with rare diseases often becomes a prolonged odyssey,…
Despite advances in research and the advent of multiple investigational treatments, pancreatic cancer is still incredibly difficult to treat. The five-year survival rate sits at just 12%. Existing therapies…
Actress Francia Raisa is well-known for her roles in Grown-Ish, Bring It On: All Or Nothing, The Secret Life of the American Teenager, and How I Met Your Father. She’s…
When Camille Wahl was a child, she loved Irish step dance. She translated her love physically, becoming a competitive dancer until she was ten years old. It was then, reports…
The Cholangiocarcinoma Foundation has recently released a white paper titled “Cross-over arms in oncology clinical trials: An important consideration for patients.” This paper highlights a non-traditional clinical trial approach that…
Recent results are available from the dose escalation portion of the Phase 1 ANTLER study. In reporting from Targeted Oncology, Jordyn Sava shares that the study included 16 participants living…
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