Search results
Results From The WOW.Com Content Network
Krabbe disease (globoid cell leukodystrophy) Mucopolysaccharidoses. Hurler syndrome (MPS I H, α-L-iduronidase deficiency) Scheie syndrome (MPS I S) Hurler–Scheie syndrome (MPS I H-S) Hunter syndrome (MPS II, iduronidase sulfate deficiency) Sanfilippo syndrome (MPS III) Morquio syndrome (MPS IV) Maroteaux–Lamy syndrome (MPS VI) Sly syndrome ...
Rett syndrome (RTT) is a genetic disorder that typically becomes apparent after 6-18 months of age and almost exclusively in girls. [4] Symptoms include impairments in language and coordination, and repetitive movements. [ 4 ]
The results of a phase-III trial, the Autologous Stem Cell Transplantation International Scleroderma (ASTIS) trial, with 156 patients, were published in 2014. HSCT itself has a high treatment mortality, so in the first year, the survival of patients in the treatment group was lower than the placebo group, but at the end of 10 years, the ...
Graft-versus-host disease (GvHD) is a syndrome, characterized by inflammation in different organs. GvHD is commonly associated with bone marrow transplants and stem cell transplants. White blood cells of the donor's immune system which remain within the donated tissue (the graft) recognize the recipient (the host) as foreign (non-self). The ...
Edmonds, the oldest potential cure case at 63 when he received a stem-cell transplant for AML in 2019, received reduced-intensity chemotherapy because of his age. Off antiretrovirals since March ...
In 2007, a team of doctors in Berlin, Germany, including Gero Hütter, performed a stem-cell transplant for leukemia patient Timothy Ray Brown, who was also HIV-positive. [79] From 60 matching donors, they selected a [CCR5]-Δ32 homozygous individual with two genetic copies of a rare variant of a cell surface receptor. This genetic trait ...
GW Pharmaceuticals is running a 252-subject Phase 3 clinical trial in 2019 with Epidiolex (CBD) in an attempt to treat Rett syndrome. [ 21 ] Trofinetide is a novel synthetic analog of the amino‐terminal tripeptide of IGF-1 designed to treat the core symptoms of Rett syndrome by reducing neuroinflammation and supporting synaptic function, it ...
Hematopoietic stem cell transplant Treatment of Wiskott–Aldrich syndrome depends on the severity of the disease. WAS is primarily a disorder of the blood-forming tissues, so in cases of severe disease (WAS score 3–5) the only widely available curative treatment currently available is a hematopoietic stem cell transplant (HCT).