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1 month ago | 
cushingsdiseasenews.com | Marisa Wexler |Shayna Korol |Andrea Lobo |Patricia Inacio
Sterotherapeutics has announced the launch of a Phase 2 clinical trial to test its experimental therapy ST-002 in people with Cushing’s syndrome. “This trial represents a significant step forward in our mission to develop transformative therapies for unmet medical needs,” Manohar Katakam, PhD, CEO of Sterotherapeutics, said in a company press release. The study’s goals, according to Sterotherapeutics, will be to evaluate the safety, tolerability, and effectiveness of ST-002 in Cushing’s patients.
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Jan 10, 2025 | 
epidermolysisbullosanews.com | Steve Bryson |Shayna Korol |Margarida Maia |Patricia Inacio
Becoming a self-taught provider of home-based skin care is one of the many challenges faced by parents of children with epidermolysis bullosa (EB) — most of whom reported often being the primary caregiver for their affected sons and daughters in an interview-based study from Norway. Other challenges identified in the new study were balancing the roles of parent and healthcare provider, and acting as gatekeepers to avoid potential EB-related problems to protect their child’s well-being.
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Nov 29, 2024 | 
praderwillinews.com | Steve Bryson |Lindsey Shapiro |Andrea Lobo |Shayna Korol
Hospitalizations among people with Prader-Willi syndrome (PWS) are characterized by higher healthcare utilization and complexity compared with non-PWS patients, according to a large-scale analysis of a U.S.-based administrative claims database. In addition to high rates of obesity, PWS patients had longer hospital stays, higher associated costs, and more in-hospital deaths. Among the youngest PWS children, the disease itself and respiratory failure drove most hospitalizations.
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Nov 13, 2024 | 
charcot-marie-toothnews.com | Marisa Wexler |Alice Melão |Yedida Y Bogachkov |Shayna Korol
A team of U.S. researchers is calling for routine screening for hip dysplasia — when the bones in the hip joint don’t fit together correctly, causing instability — among children with Charcot-Marie-Tooth disease (CMT), after their work revealed it to be a common but underrecognized problem in young people with the inherited condition.
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Nov 7, 2024 | 
alsnewstoday.com | Esteban Cerezo |Mary Chapman |Shayna Korol |Dagmar Munn
The government of Ontario has committed CA$13 million (around $9.36 million) over three years to provide critical and comprehensive care to people with amyotrophic lateral sclerosis (ALS) who live in the Canadian province. The funding, which was announced in Ontario’s 2024 Fall Economic Statement, will help support the new Ontario Provincial ALS Program meet the needs of the roughly 1,400 residents of Ontario living with ALS.
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Oct 23, 2024 | 
myastheniagravisnews.com | Andrea Lobo |Shayna Korol |Lindsey Shapiro |Mary Chapman
The new UCB Myasthenia Gravis Scholarship, launched by the Belgium-based biopharmaceutical company, is aiming to help myasthenia gravis (MG) patients and their family members in the U.S. to continue their education or learn new skills by covering educational expenses — with program awards totaling up to $150,000.
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Aug 13, 2024 | 
huntingtonsdiseasenews.com | Marisa Wexler |Marta Costa Figueiredo |Steve Bryson |Shayna Korol
Being physically active is significantly associated with a delayed age of the onset of Huntington’s disease and spending more time watching television is significantly linked to an earlier age of onset. These are the findings of a study that analyzed genetic variants linked with different levels of physical activity and sedentary behaviors, and the possible associations with differences between expected age of onset (based on genetic data) and actual age of onset.
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May 15, 2024 | 
ancavasculitisnews.com | Margarida Maia |Andrea Lobo |Shayna Korol
Argenx has decided not to go ahead with its development plans for efgartigimod as a potential treatment for ANCA-associated vasculitis (AAV) after the therapy failed to show beneficial effects in late-stage clinical trials of two other autoimmune diseases. Efgartigimod is approved in the U.S. in two formulations for treating adults with generalized myasthenia gravis, an autoimmune disease that causes muscle weakness.
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Apr 11, 2024 | 
epidermolysisbullosanews.com | Patrice C. Williams |Marisa Wexler |Patricia Inacio |Shayna Korol
Earlier this week, my son Jonah and I had a visit with his pain doctor, Dr. Savi. She is the coolest, even in the eyes of the ultimate coolness judge, a 15-year-old boy. She oohs and aahs appropriately over Jonah’s growth. I tell her Jonah is now 5 feet, 2 inches tall, and Jonah corrects me by saying he’s 5 feet, 3 inches tall. Dr. Savi agrees with Jonah, as any cool doctor would, and congratulates him on his added inches and deepening voice.
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Apr 5, 2024 | 
cushingsdiseasenews.com | Marisa Wexler |Mary Chapman |Shayna Korol
Cushing’s Awareness Day is April 8, and the Cushing’s community is gearing up with events and fundraising efforts to spread awareness and raise money to combat the disorder. In celebration of this year’s Awareness Day, Xeris Pharmaceuticals, which markets Cushing’s treatment Recorlev (levoketoconazole), is teaming up with the Magic Foundation, a nonprofit focused on helping people with hormonal disorders, to host a virtual town hall meeting.
