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Dec 5, 2024 | 
cysticfibrosisnewstoday.com | Steve Bryson |Marisa Wexler |Joana Vindeirinho |Joana Carvalho
Researchers discovered a molecule that may help deliver gene therapies to cells in the thick mucus environment of the cystic fibrosis (CF) lung, a study reported. Since thick mucus makes gene delivery to the CF lung a significant challenge, “we are encouraged by these results and anticipate that our [molecule] could be used to successfully deliver cystic fibrosis gene therapies in future work,” the authors wrote.
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Nov 25, 2024 | 
copdnewstoday.com | Margarida Maia |Joana Vindeirinho |Caroline Gainer |Steve Bryson
Oral supplementation with nicotinamide riboside, a form of vitamin B3, helped reduce markers of lung inflammation in older adults with chronic obstructive pulmonary disease (COPD), data from a placebo-controlled study shows. The benefits were sustained for at least three months after supplementation ended.
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Oct 18, 2024 | 
parkinsonsnewstoday.com | Marisa Wexler |Joana Vindeirinho |Lindsey Shapiro |Steve Bryson
The U.S. Food and Drug Administration (FDA) has approved foslevodopa and foscarbidopa, a levodopa therapy given as a 24-hour under-the-skin infusion, to treat motor fluctuations in adults with advanced Parkinson’s disease. The newly approved therapy, which had been known as ABBV-951 while in development, will be sold by its developer AbbVie under the brand name Vyalev, the company said in a press release announcing the FDA decision.
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Sep 30, 2024 | 
copdnewstoday.com | Margarida Maia |Joana Vindeirinho |Lindsey Shapiro |Marisa Wexler
The U.S. Food and Drug Administration (FDA) has approved Dupixent (dupilumab) as an add-on maintenance treatment for adults with poorly controlled chronic obstructive pulmonary disease (COPD) and high counts of immune cells called eosinophils. Elevated eosinophils are a marker of type 2 inflammation, which is thought to contribute to COPD-related lung inflammation. The approval comes after the FDA delayed its decision by three months to give regulators enough time to complete their review.
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Aug 12, 2024 | 
copdnewstoday.com | Marisa Wexler |Joana Vindeirinho |Caroline Gainer |Steve Bryson
The U.S. Food and Drug Administration (FDA) has granted breakthrough designation to tezepelumab, an approved therapy for severe asthma, as an add-on maintenance treatment for moderate to very severe chronic obstructive pulmonary disease (COPD) marked by an eosinophilic profile. An eosinophilic profile refers to high blood levels of eosinophils, a type of immune cell.
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Aug 6, 2024 | 
hemophilianewstoday.com | Esteban Cerezo |Joana Vindeirinho |Margarida Maia |Alliah Czarielle
Prophylactic treatment for hemophilia can help people with the disease avoid unemployment due to disability, a study reports. Hemophilia has a negative affect on a person’s employability.
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Jul 22, 2024 | 
ancavasculitisnews.com | Andrea Lobo |Steve Bryson |Joana Vindeirinho |Patricia Inacio
Using benralizumab — a medication approved in the U.S. for a rare type of asthma — off-label as an add-on to standard induction treatment resulted in a high rate of disease remission in people with eosinophilic granulomatosis with polyangiitis (EGPA) and severe asthma.
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May 3, 2024 | 
multiplesclerosisnewstoday.com | Steve Bryson |Joana Vindeirinho |Margarida Maia |Lindsey Shapiro
Charles River Laboratories has joined up with FibroBiologics to manufacture the company’s spheroids for clinical trials in multiple sclerosis (MS) and several other conditions. The spheroids are groups of fibroblast cells arranged in a three-dimensional (3D) structure that more closely resemble how cells organize in living tissues. Fibroblasts are abundant in connective tissue and can regenerate damaged tissues, including nerve cell damage in MS.
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May 1, 2024 | 
pulmonaryhypertensionnews.com | Andrea Lobo |Joana Vindeirinho |Margarida Maia |Patricia Inacio
Low levels of PM20D1, a molecule involved in metabolizing lipids, were identified as a risk factor for mortality in people with pulmonary arterial hypertension (PAH), a study reports. As a result, PM20D1, or peptidase M20 domain containing 1, may be a biomarker of disease prognosis, according to scientists. Lipid metabolism is a complex process that includes the production, intake, degradation or conversion of fatty molecules.
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Mar 22, 2024 | 
smanewstoday.com | Patricia Inacio |Marisa Wexler |Joana Vindeirinho |Marta C. Figueiredo
Scholar Rock remains on track to report topline results later this year from its ongoing Phase 3 clinical trial of apitegromab in children and young adults with spinal muscular atrophy (SMA) types 2 or 3. Apitegromab is an antibody that targets myostatin, a protein involved in suppressing muscle growth. This should boost muscle strength and improve patients’ motor function.
