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1 month ago | 
musculardystrophynews.com | Lindsey Shapiro |Marisa Wexler |Patricia Inacio |Margarida Azevedo
Capricor Therapeutics’ investigational therapy deramiocel, formerly CAP-1002, which is being considered for approval in the U.S., continued to show an ability to preserve upper limb function in boys and men with Duchenne muscular dystrophy (DMD) over the long term. That’s according to analyses of about five years worth of data spanning the Phase 2 HOPE-2 trial (NCT03406780), its open-label extension study (NCT04428476), and a gap period without treatment.
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Jan 15, 2025 | 
alsnewstoday.com | Dagmar Munn |Esteban Cerezo |Juliet Taylor |Margarida Azevedo
Do you have an ALS “inspiration folder?” I do. And I just added another name to my ever-growing list. Even though most people in my folder don’t have ALS, what inspires me is that despite their dire circumstances, they dare to have goals and find ways to keep moving forward. Knowing their stories and the challenges they face helps me power through the days when I feel like I’m in an uphill battle with my ALS symptoms.
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Aug 8, 2024 | 
sicklecellanemianews.com | Marisa Wexler |Dunstan Nicol-Wilson |Joana Fernandes |Margarida Azevedo
A bone marrow transplant may help reverse heart damage in people with sickle cell disease (SCD), according to a study. The findings were published in a letter to the editor titled, “Impact of Hematopoietic Cell Transplantation on Myocardial Fibrosis in Young Patients with Sickle Cell Disease,” in Blood. SCD is a genetic disorder characterized by the presence of abnormally shaped red blood cells.
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Jul 19, 2024 | 
cushingsdiseasenews.com | Andrea Lobo |Lindsey Shapiro |Margarida Azevedo |Patricia Inacio
Mutations in the USP8 gene are found in 35% of the pituitary corticotroph adenomas that cause Cushing’s disease, and more commonly affect women than men, a review study found. Thus, targeting the gene and epidermal growth factor receptor (EGFR) — whose levels are increased in patients with USP8 mutations — may be a promising therapeutic approach for these patients.
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Jan 29, 2024 | 
fapnewstoday.com | Margarida Maia |Margarida Azevedo |Patricia Inacio |Marisa Wexler
New 15-year data from a global study involving more than 6,000 people with ATTR amyloidosis, an umbrella term for conditions that include familial amyloid polyneuropathy (FAP), found that nearly 1 in 4 patients experience both neurological and cardiac symptoms, showing the need, according to researchers, for improved multidisciplinary disease management.
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Oct 9, 2023 | 
lupusnewstoday.com | Marisa Wexler |Margarida Azevedo |Marta Costa Figueiredo |Steve Bryson
In both the U.S. and Europe, clinicians want to limit treatment with glucocorticoids — steroid hormones whose long-term use for autoimmune diseases is tied to serious side effects — among people with moderate to severe lupus. To that end, healthcare professionals are increasingly prescribing biological therapies like Benlysta (belimumab) and Saphnelo (anifrolumab).
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Sep 1, 2023 | 
alsnewstoday.com | Marisa Wexler |Patricia Valerio |Lindsey Shapiro |Margarida Azevedo
Mutations in the C9ORF7 gene, the most common genetic cause of amyotrophic lateral sclerosis (ALS), lead to defects in the stem cells needed for brain development in the early stages of life, a new study shows. In animal models of the disease, this resulted in smaller than normal volumes of certain brain regions even before birth. “We are only beginning to understand how neurodevelopment in an embryo can contribute to neurodegeneration in an adult.
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Aug 29, 2023 | 
sicklecellanemianews.com | Mary Chapman |Margarida Azevedo
To help bring new therapies more quickly and cost effectively to those with sickle cell disease (SCD) in sub-Saharan countries — where patients bear a significantly high SCD burden — the U.S.-based biotechnology company Functional Fluidics is planning to expand its operations into Africa.
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May 18, 2023 | 
sicklecellanemianews.com | Steve Bryson |Margarida Azevedo |Marisa Wexler |Andrea Lobo
Lobe Sciences has partnered with Pentec Health to distribute Altemia, an emulsified formulation of docosahexaenoic acid (DHA), a type of omega-3 fat, for sickle cell disease (SCD) patients in the U.S. and its territories. As part of the agreement, Pentec, which provides clinical nutrition and specialty care services in the U.S., will receive the exclusive rights to distribute the medical food, which is expected to be available to adults and children with SCD by the second half of this year.
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Feb 13, 2023 | 
fapnewstoday.com | Marisa Wexler |Margarida Azevedo |Somi Igbene
Most people with late-onset familial amyloid polyneuropathy (FAP) experience pain as a result of nerve damage, referred to as neuropathic pain, a recent study highlights. Findings suggest that neuropathic pain is linked to worse life quality, and many patients do not experience relief from this type of pain even with treatment. Researchers stressed that it’s important for clinicians to address pain in people with FAP.
