Nonprofit Group Team Joseph Helps Meet the Needs of Patients With DMD
The nonprofit Team Joseph raises money for scientific studies, but also looks for immediate gaps or needs not being met by other patient organizations.
Pulmonologists
An Interview With Marissa Penrod, Founder of DMD Nonprofit Group Team Joseph
Larry Luxner, senior correspondent for Rare Disease Advisor, interviews Marissa Penrod, founder of the Indiana-based nonprofit group Team Joseph.
Fazirsiran Improves Liver Enzyme Concentration in AATD Trial
Fazirsiran is associated with a notable reduction in the concentration of Z-AAT, the most common form of alpha-1 antitrypsin (AAT) causing AAT deficiency (AATD), in the serum and liver as well as with improvements in the concentrations of liver enzymes, according to the results of a phase 2 clinical trial published in the New England…
Valproic Acid Affects Efficacy of Nusinersen in SMA Study
The histone deacetylase inhibitor valproic acid cooperates with nusinersen-like antisense oligonucleotides to promote the inclusion of exon 7 and transcriptional elongation from the SMN2 gene, which means that more functional SMN protein can be made from the SMN2 gene. This approach could increase the efficacy of nusinersen and other antisense oligonucleotides for the treatment of…
Combinatorial Therapy Fails to Counteract SMA Pathology
Scientists have reported that a combinatorial therapy of antisense oligonucleotide (ASO) mediated therapy and low dose SMN with the protective modifier Chp1 is unable to significantly decrease the key pathological features of spinal muscular atrophy (SMA), as published in the Neurobiology of Disease. SMA is a progressive neurodegenerative disorder that often results in early death.…
One Session of Autogenic Drainage Reduces Peripheral Resistance in CF Study
One session of autogenic drainage can enhance peripheral clearance in children with cystic fibrosis (CF), particularly those with bronchial congestion, according to a study published in BMC Pulmonary Medicine. The researchers noted that the technique did not modify peripheral resistance in children without congestion. “In a long-term comparative study in adolescents with CF, autogenic drainage…
Three Lakes Foundation and CHEST Collaborating to Speed Up Diagnosis of IPF
A pulmonary fibrosis foundation and a professional medical association have teamed up to slash the time it takes to diagnose idiopathic pulmonary fibrosis.
Dr. Mary Beth Scholand Discusses CHEST Partnership With the Three Lakes Foundation
Rare Disease Advisor senior correspondent interviews Mary Beth Scholand, MD, director of the Interstitial Lung Disease Program at the University of Utah in Salt Lake City.
Study Sheds Light on Pathology of End-Stage Lung Disease in IPF and CF
Adaptive and innate lymphocyte populations contribute to interleukin (IL)-17A-dependent pathologies in the end-stage of chronic lung diseases such as idiopathic pulmonary fibrosis (IPF) and cystic fibrosis (CF), according to a new study published in Clinical and Translational Immunology. The study also showed that pathogens drive the secretion of IL-17A. IL-17A and IL-22 play an important…
BOS-318 Inhibitor Shows Mutation-Agnostic Properties in CF
The use of BOS-318 in human ex vivo models has shown an increase in airway hydration, mucociliary clearance, and protection against epithelial cell death due to Pseudomonas aeruginosa infection, therefore suggesting a potential therapeutic value in cystic fibrosis (CF), as published in Cell Chemical Biology. The pathogenesis of CF is associated with an epithelial sodium channel…
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