Nature10y
Conformational disease
The recent conference, `Alpha 1-antitrypsin deficiency and other conformational ... Michigan) described the phenotype of a mouse `knock-in' of a mutant allele of eIF2α that cannot be ...
Korro Bio’s treatment of alpha-1 antitrypsin deficiency gets orphan designation
Korro Bio’s (KRRO) treatment of alpha-1 antitrypsin deficiency received FDA orphan designation, according to a post on the agency’s stie.Light ...
Medscape2mon
Autoimmune Hepatitis -- Approach to Diagnosis
alpha-1 AT = alpha-1 antitrypsin; ANA = antinuclear antibodies; SMA = smooth muscle antibodies; anti-LKM1 = antibodies to liver/kidney microsome type 1; AMA = antimitochondrial antibodies ...
FierceBiotech10d
Beam gene therapy for protein deficiency passes early safety test, sets efficacy bar in space: analyst
The one-time therapy appears to correct the mutation that causes the disease, known as Alpha-1 antitrypsin deficiency (AATD), with a higher dose leading to more functional molecules of the protein ...
Yahoo Finance6d
KRRO-110 Receives Orphan Drug Designation from U.S. FDA for Alpha-1 Antitrypsin Deficiency
has granted orphan drug designation to the investigational medicine KRRO-110 for the treatment of Alpha-1 Antitrypsin Deficiency (AATD). “Receiving orphan drug designation from the FDA ...
Medscape3d
Augmentation Therapy Reduces Frequency of Lung Infections in Antitrypsin Deficiency: A New Hypothesis With Supporting Data
Design: The proposed concept is based on a survey taken via the Internet of patients receiving augmentation therapy for 1 ... phenotype for AAT deficiency to determine whether those receiving ...
Hosted on MSN1mon
Wave Life Sciences begins Phase 1 obesity trial
The drug aims to induce a healthy phenotype associated with less abdominal ... has a diverse pipeline that includes programs for Alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy ...