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 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 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…
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…
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…
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…
In early stages, renal cell carcinoma (RCC) is treatable and has a high five-year survival rate. However, if the cancer progresses or metastasizes (spreads), the five-year survival rate falls…
Prior to February 2023, there were no FDA-approved treatment options for non-central nervous system manifestations of alpha-mannosidosis. But in mid-February 2023, Chiesi Global Rare Diseases shared via news release…
Currently, an estimated 47-50% of global nasopharyngeal carcinoma cases occur in China, with the regions of Guangdong, Guangxi and Fujian being heavily affected. There are a number of treatment…
In a news release from commercial-stage biopharmaceutical company Calliditas Therapeutics AB CALT ("Calliditas"), the company shared positive topline results from the Phase 3 NefIgArd study. Within the study, researchers…
According to a story on pharmaphorum.com, the UK's National Institute for Health and Care Excellence (NICE) has given a positive opinion for the drug vutrisiran (marketed as Amvuttra) as a…
The Orphan Drug Act was passed with a goal in mind: to incentivize the development of therapeutics for orphan drugs. These drugs are used for patients with rare conditions,…
Shifts in treatment paradigms can improve the lives of countless patients and contribute to better overall outcomes. In a news release from February 23, 2023, Sanofi and international biopharmaceutical…
According to a story from World Pharma Today, Andelyn Biosciences, Inc., which is a gene therapy Contract Development and Manufacturing Organization (CDMO), has recently struck a partnership with Odylia Therapeutics,…
Until recently, the only therapeutic options for people with Friedreich ataxia were symptomatic and focused on symptom management: mobility aids, surgery, psychotherapy, speech therapy, heart medications, hearing and vision…
According to a story from Globe Newswire, the gene therapy company uniQure recently announced that it has received conditional marketing authorization for its gene therapy etranacogene dezaparvovec (marketed as HEMGENIX®).…
In the United States, Orphan Drug designation is granted to drugs or biologics designed to treat, prevent, or diagnose rare conditions. “Rare” conditions refer to those affecting under 200,000…
Researchers have been grappling for years with the STAT5 protein as a tool to fight cancer. STAT5 is critical because it is partly responsible for the development and progression…
Proteinuria refers to excess protein in the urine. In healthy individuals, proteins pass through the kidneys and into the bloodstream. Proteinuria often indicates kidney dysfunction or damage. In fact,…
Currently, a glioblastoma multiforme (also known simply as glioblastoma or GBM) diagnosis comes with a poor prognosis. There is a 40% survival rate after one year which falls to…
Dr. Seth Lederman founded Tonix Pharmaceuticals with a deep desire to change the treatment landscape for patients. The company’s rich pipeline features therapeutic options for fibromyalgia, post-traumatic stress disorder (PTSD),…
The advocacy group Just Treatment recently published a press release titled 'BREAKING: Cystic Fibrosis Patients Launch Global Challenge to Vertex Monopoly on CF Drugs.' Vertex Pharmaceuticals has taken the lead…
Two Southern California University studies showed a reduction of neurodegenerative activity in amyotrophic lateral sclerosis (ALS) research models with each using a different treatment route. As reported in Inside Precision…
For many people living with mantle cell lymphoma (MCL), the standards-of-care include Bruton's tyrosine kinase (BTK) inhibitors. But there is a subset of patients who do not respond well,…
PART I The baby died almost immediately after birth. Edwards Syndrome is a rare congenital disease. Only about ten percent of newborn babies survive past their first year. Babies…
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