Aplastic Anemia News and Research

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Gene editing technology shines light on rare dyskeratosis congenita

Using the gene editing technology CRISPR, scientists have shed light on a rare, sometimes fatal syndrome that causes children to gradually lose the ability to manufacture vital blood cells.

27 Jul 2017

Latinos left out of clinical trials … and possible cures

Two decades ago, Luis Antonio Cabrera received devastating news: He likely had only three months to live.

19 Jul 2017

FDA approves expanded use of systemic therapy for treating chronic moderate-to-severe pediatric psoriasis

Amgen today announced that the U.S. Food and Drug Administration has approved the supplemental Biologics License Application for the expanded use of ENBREL (etanercept), making it the first and only systemic therapy to treat pediatric patients (ages 4-17) with chronic moderate-to-severe plaque psoriasis.

7 Nov 2016

Alexion initiates new global Uncommon Strength campaign to raise awareness of rare diseases

Alexion Pharmaceuticals, Inc. today announced the launch of Uncommon Strength, a global campaign to raise awareness of rare diseases through the celebration of the extraordinary resilience and inner strength of those impacted by these diseases.

15 Sep 2016

Male hormones can reverse biological drivers of aging, study shows

Telomerase, an enzyme naturally found in the human organism, is the closest of all known substances to a "cellular elixir of youth." In a recent study, Brazilian and US researchers show that sex hormones can stimulate production of this enzyme.

26 Jul 2016

Scientists discover new way to predict risk for delayed recovery in children with AML

The chemotherapy treatments necessary to treat Acute Myeloid Leukemia (AML) in children can be grueling on the body, and can cause health-related complications during therapy, as well as long down the road after remission.

1 Jul 2016

Musashi-2 protein regulates function, development of blood stem cells

Researchers at McMaster University's Stem Cell and Cancer Research Institute have made significant steps forward in understanding the stem cells of the human blood system after discovering how a key protein allows for better control and regeneration of these cells.

28 Apr 2016

New anti-ageing therapy may be effective against aplastic anaemia

Aplastic anaemia is a rare, and potentially fatal, disease of the blood, by which the bone marrow is unable to generate blood cells at the appropriate pace. Many forms of aplastic anaemia share an important link with the ageing process: the shortening of telomeres, structures that protect the ends of chromosomes.

25 Feb 2016

Mylan releases generic version of Felbatol Tablets in U.S.

Mylan N.V. today announced the U.S. launch of Felbamate Tablets USP, 400 mg and 600 mg, which is the generic version of Meda Pharms' Felbatol Tablets. Mylan received final approval from the U.S. Food and Drug Administration for its Abbreviated New Drug Application (ANDA) for this product.

19 Jan 2016

SCCA's Fred Hutch Bone Marrow Transplant Program recognized for outstanding survival rates

The Fred Hutch Bone Marrow Transplant Program at Seattle Cancer Care Alliance has earned recognition by the Center for International Blood and Marrow Transplant Research for outperforming its expected one-year survival rates for allogeneic transplant patients - those who receive donated adult blood-forming stem cells.

23 Dec 2015

FDA approves Promacta to treat pediatric patients with chronic immune thrombocytopenic purpura

The U.S. Food and Drug Administration today approved Promacta (eltrombopag) to treat low blood platelet count in pediatric patients – ages one year and older – with a rare blood disorder called chronic immune thrombocytopenic purpura (ITP). Promacta can be used in these children when they have not achieved an appropriate response using other ITP medicines or surgery to remove the spleen.

9 Oct 2015

Regen BioPharma develops novel means of delivering RGBP-248 gene silencing nucleic acid to liver tumors

Regen BioPharma, Inc. announced today development of a novel means of delivering its patented (US Patent 8,263,571) gene silencing nucleic acid, RGBP-248, to liver tumors utilizing a clinically approved formulation as delivery system. The product in development overcomes previous hurdles with gene silencing therapeutics in which the gene silencing drug had to be delivered throughout the whole body.

31 Aug 2015

Regen BioPharma implements gene silencing technology in killing cancer stem cells

Regen BioPharma Inc. reported today the successful implementation of its gene silencing technology, covered by patent # 8,263,571, in killing cancer stem cells.

24 Aug 2015

Study of genetic mutations could lead to optimized treatment plans for aplastic anemia patients

Scientists have identified a group of genetic mutations in patients with aplastic anemia, which likely will help doctors optimize treatment for this rare and deadly blood condition. The study, appearing in the New England Journal of Medicine, could lead to tailor-made treatment plans for aplastic anemia patients as part of the emerging precision medicine movement.

2 Jul 2015

MSD announces EC approval of SIMPONI (golimumab) for treatment of adult patients with nr-axial SpA

MSD (Merck & Co., Inc., Kenilworth, New Jersey, USA) today announced that on 22 June the European Commission approved SIMPONI (golimumab) for the treatment of adult patients with severe, active non-radiographic axial spondyloarthritis (nr-axial SpA).

26 Jun 2015

UCB sponsoring several presentations on Cimzia for Crohn's disease at DDW 2015

UCB, a global biopharmaceutical company focusing on immunology and neurology treatment and research, is sponsoring several data presentations on Cimzia (certolizumab pegol) at Digestive Disease Week 2015, taking place in Washington, DC from May 16-19.

17 May 2015

Clinical trial uses personalized cellular therapy to treat GVHD complication of bone marrow transplant

An innovative clinical trial using the science of "personalized" cellular therapy has begun enrolling children and adults suffering from graft-versus-host-disease (GVHD), a life-threatening complication of bone marrow transplantation in which donor immune lymphocytes attack the organs of the bone marrow transplant recipient.

3 Apr 2015

Mount Sinai researchers reprogram blood cells into iPSCs to study genetic origins of MDS

Induced pluripotent stem cells (iPSCs) -- adult cells reprogrammed back to an embryonic stem cell-like state--may better model the genetic contributions to each patient's particular disease. In a process called cellular reprogramming, researchers at Icahn School of Medicine at Mount Sinai have taken mature blood cells from patients with myelodysplastic syndrome (MDS) and reprogrammed them back into iPSCs to study the genetic origins of this rare blood cancer.

26 Mar 2015

Longer donor leukocyte telomere length linked to improved survival following HCT

Among patients with severe aplastic anemia who received stem cell transplant from an unrelated donor, longer leukocyte (white blood cells) telomere length (a structure at the end of a chromosome) was associated with increased overall survival at 5 years, according to a study in the February 10 issue of JAMA.

12 Feb 2015

SCCA's Fred Hutch Bone Marrow Transplant Program achieves higher survival rates

The Fred Hutchinson Cancer Research Center Bone Marrow Transplant Program at Seattle Cancer Care Alliance was recently recognized for outperforming its anticipated one-year survival rate for allogeneic transplant patients.

28 Jan 2015