According to a recent article in MedCity News, families with children who have a rare disease wait on average for five years before receiving an accurate diagnosis. It is…
In early May 2021, pharmaceutical company GlaxoSmithKline shared that its therapy Benlysta (belimumab) was approved by the European Commission (EC) for expanded use in adult patients with lupus nephritis…
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…
As the pandemic closed bars and theaters and so many of our jobs became remote, much of the world became virtual. And as cocktail hour moved onto zoom and museums…
Rare disease research faces many obstacles: lack of participants, lack of funding, and lack of interest are just a few. Now, the charity Cerebra has launched a network to take…
Medscape reported that when Victoria Gray was three months old, her family was told to keep her close: her prognosis wasn't looking good. She had been rushed to the ER…
Noémie has four wonderful, healthy children. She has made it clear that amyotrophic lateral sclerosis (ALS) is not the end for her and that she values every precious moment…
During the American Society of Gene & Cell Therapy (ASGCT) 24th Annual Meeting, which took place virtually from May 11-14, 2021, biotechnology company M6P Therapeutics ("M6P") presented preclinical data…
Lori Dunham’s recent article for the Lambert-Eaton News is a message to parents that no matter what disease their children may have, teaching them to advocate for themselves is…
Though myalgic encephalomyelitis or chronic fatigue syndrome (CFS) is more widespread than many people realize, the condition has few resources and no remedy. The disabling condition is complex, with no…
PTC Therapeutics has just announced data from the very first study which examined how taking care of a child diagnosed with aromatic L-amino acid decarboxylase (AADC) deficiency can impact caregivers.…
Mustang Bio, a biopharmaceutical company, and City of Hope, a cancer center, have just revealed that their first participant in their Phase 1 clinical trial for leptomeningeal brain tumors including…
COVID-19 long-haulers, now called Post-Acute Sequelae of SARS-CoV-2 Infection, or PASC, have often been compared to myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS). However, there wasn't a study directly comparing these two…
NLS Pharma has just announced new data which further supports the mechanism of action of Mazindol. Mazindol is the active ingredient in Quilience, a treatment used for narcolepsy. This therapy…
Head and neck squamous cell carcinoma (SCC) is considered both aggressive and difficult to treat. In fact, doctors estimate that nearly 50% of all patients will eventually see the recurrence…
Recently, the FDA granted Priority Review status to a supplemental New Drug Application (sNDA) for zanubrutinib, MPR reports. While the treatment, under the brand name Brukinsa, is currently indicated for…
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…
To this day, Colleen Sosinski still isn't quite sure how her journey with necrotizing fasciitis began. During summer 2020, she was relaxing and enjoying the sun. Then, as reported by…
The CDC notes that in 2019, an estimated 409,000 people globally died of malaria - but a majority of those who died were children in sub-Saharan Africa. In fact, 94%…
For patients and families, finding a supportive community can be extremely helpful. These communities spur research, offer a sense of understanding, and build awareness. This is exactly what Susan G.…
The month of May is recognized as PKU Awareness Month, a time of year set aside to focus on spreading awareness about phenylketonuria (PKU) among the general public and in…
Compassion [kuhm-pash-uhn] noun A feeling of deep sympathy and sorrow for another who is stricken by misfortune, accompanied by a strong desire to alleviate the suffering. Compassion Corner is a…
According to a story from yahoo.finance.com, the medical technology company iCAD, Inc. recently held a webinar featuring several brain tumor experts that discussed the potential of an approach called intraoperative…
From May 11-14, 2021, the American Society of Gene and Cell Therapy (ASGCT) held its 24th Annual Meeting. Due to COVID-19, the meeting was held virtually - but that didn't…
According to a story from republicworld.com, the medical fraternity of Queen Mary's Hospital in King George's Medical University, Lucknow was able to successfully treat a woman, age 35, with immune…
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