The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Cabaletta Bio’s cell therapy CABA-201 as a treatment for adults with hard-to-treat systemic sclerosis (SSc), or scleroderma. Orphan drug designation is intended to support the accelerated development of investigational treatments for rare diseases, defined as…
More conversations, either one on one or in groups via telephone or virtually, along with in-person household activities and conversations, were significantly associated with less loneliness during the COVID-19 pandemic among people with systemic sclerosis (SSc), a study indicates. Conversations and activities at the…
Dosing has begun in a Phase 2 clinical trial evaluating aTyr Pharma’s efzofitimod, a first-in-class therapy for people with interstitial lung disease (ILD) associated with systemic sclerosis (SSc) — together known as SSc-ILD. Recruitment for the proof-of-concept EFZO-CONNECT study (NCT05892614), which aims to enroll up to 25…
The U.S. Food and Drug Administration (FDA) has cleared Cabaletta Bio’s request to begin clinical testing of CABA-201, a cell therapy for adults with hard-to-treat systemic sclerosis (SSc). Cabaletta now plans to launch a Phase 1/2 clinical trial, enrolling SSc patients with severe skin manifestations or organ…
The levels of two microRNAs are found altered in the bloodstream of adults with systemic sclerosis (SSc) compared with healthy people, according to a new study that suggests using these molecules as biomarkers of the disease. Elevated levels of one of them, called miR-21, is associated with scar formation,…
People with systemic sclerosis (SSc) have a wide range of support and information needs, regardless of their disease duration, a survey found. Patients with less time since diagnosis had greater needs overall and in areas of learning how to manage their condition and how to cope psychologically and socially.
Bloodstream levels of a protein that plays a role in the body’s response to low oxygen may be an effective biomarker to assess abnormal changes in small blood vessels associated with systemic sclerosis (SSc), according to a recent study. Levels of the protein, called hypoxia-inducible factor-1 (HIF-1), were significantly…
Plasma exchange helped reduce high blood pressure linked to scleroderma renal crisis (SRC) — a potentially life-threatening complication marked by abrupt blood pressure changes and acute kidney failure — in patients unresponsive to standard treatment, a case series study reported. Despite these successes, most patients still had impaired kidney…
Elevated activity of a blood-clotting protein called Factor XIII (FXIII) in the bloodstream of adults with systemic sclerosis (SSc) is tied to the presence of Raynaud’s phenomenon, a common SSc symptom, a small study suggests. While most study participants had FXIII activity levels within the normal range, “patients with…
Caregivers of children with juvenile scleroderma cited a lack of knowledge about the rare disease within the medical community as the primary barrier to a proper diagnosis and treatment, according to a new survey study. For more than a quarter of the children, it took more than a year…
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