As the name implies, a rare disease is an illness that affects far fewer people than more common conditions, like breast cancer or heart disease. Today, however, 7,000 rare conditions have been ...
Please provide your email address to receive an email when new articles are posted on . Researchers analyzed 19 children with Pompe disease seen at a Texas-based children’s hospital.
In the United States about 5%-8% of the population is affected by an autoimmune disorder. Other reports, however, estimate a higher prevalence of various types of autoimmune disorders due to complex ...
Stem cells transplanted in utero (green) engrafted into fetal mouse brain tissue. Image credit: Q-H Nguyen/MacKenzie lab. Administering stem cell or enzyme therapy in utero may be a path to ...
Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window The first test ...
Acquisition is Strong Strategic Fit for BioMarin, Adding INZ-701, a Phase 3 Enzyme Replacement Therapy Being Developed for Treatment of ENPP1 Deficiency First Pivotal Data Readout in Children Expected ...
FDA approves avalglucosidase alfa-ngpt (Nexviazyme) for the treatment of patients 1 year and older with late-onset Pompe disease. FDA announced yesterday the approval of avalglucosidase alfa-ngpt, ...
WALTHAM, Mass., June 24, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for ...
Please provide your email address to receive an email when new articles are posted on . A phase 3 trial evaluating intrapleural enzyme therapy vs. video-assisted thoracoscopic surgery is feasible.
A new study led by University of Pittsburgh and UPMC Hillman Cancer Center researchers shows that an enzyme called PARP1 is involved in repair of telomeres, the lengths of DNA that protect the tips of ...
Objective: To compare the short-term efficacy and tolerability of an oral enzyme therapy with the NSAID diclofenac in patients with symptomatic osteoarthritis of the knee (gonarthritis). Methods: In a ...
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