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Study shows pulmonary alveolar proteinosis can be cured by single transfer of monocytes

Study shows pulmonary alveolar proteinosis can be cured by single transfer of monocytes

Researchers from VIB-UGent reveal that adult circulating monocytes that get access to the macrophage niche in the liver or the lung can acquire identical tissue-specific macrophage functions and self-maintenance capacities as macrophages of embryonic origin. [More]
New therapy strategy for liver disease shows initial measureable success with norUrso

New therapy strategy for liver disease shows initial measureable success with norUrso

The new therapy strategy for primary sclerosing cholangitis, a liver disease that at present still cannot be cured with medication, shows initial measureable success with the nor-ursodeoxycholic acid (norUrso). [More]
Complete genetic map of scleroderma opens door for diagnosis and targeted treatment

Complete genetic map of scleroderma opens door for diagnosis and targeted treatment

Scleroderma is an autoimmune disease that affects one out of every 10,000 people in Europe and North America, mostly middle-aged women, and causes death in a high percentage of cases. [More]
UT Southwestern scientists discover mutation that causes X-linked reticulate pigmentary disorder

UT Southwestern scientists discover mutation that causes X-linked reticulate pigmentary disorder

UT Southwestern Medical Center researchers have discovered a mutation that causes a rare systemic disorder known as X-linked reticulate pigmentary disorder (XLPDR) and, significantly, the unexpected cellular mechanism by which the mutation causes the disease. [More]
Anthim (obiltoxaximab) injection approved for treatment of inhalational anthrax

Anthim (obiltoxaximab) injection approved for treatment of inhalational anthrax

On Friday, March 18, the U.S. Food and Drug Administration approved Anthim (obiltoxaximab) injection to treat inhalational anthrax in combination with appropriate antibacterial drugs. Anthim is also approved to prevent inhalational anthrax when alternative therapies are not available or not appropriate. [More]

New molecular probe can detect different amyloid deposits in human tissues

A team of scientists at Sweden's Linköping University have developed a molecular probe that can detect an array of different amyloid deposits in several human tissues. This new probe is extremely sensitive and was used at very low concentrations to correctly identify every positive amyloidosis sample when compared to the traditional clinical tests. [More]
Systemic sclerosis: an interview with Dr Kristin Highland

Systemic sclerosis: an interview with Dr Kristin Highland

Systemic sclerosis, also known as scleroderma, is a rare disease characterized by the thickening and scarring of connective tissue of multiple organs in the body [More]
Study identifies gene linked to rare, complex diseases

Study identifies gene linked to rare, complex diseases

IRB Barcelona identifies GEMC1 as a master gene for the generation of multiciliated cells—cells with fine filaments that move fluids and substances—which are found exclusively in the brain, respiratory tract, and reproductive system.Defects in multiciliated cells lead to ciliopathies—rare and complex diseases that are poorly understood and for which not all causative genes have been identified. [More]
New international survey provides insights into emotional truths of living with IPF

New international survey provides insights into emotional truths of living with IPF

An international survey of over 500 people living with idiopathic pulmonary fibrosis (IPF) in more than 20 countries worldwide reveals the real-life emotions faced by those with the devastating disease. [More]
Rare Disease Day 2016: Boehringer Ingelheim launches new initiatives to raise awareness of IPF

Rare Disease Day 2016: Boehringer Ingelheim launches new initiatives to raise awareness of IPF

Boehringer Ingelheim, working with pharmaphorum media, has brought together patients, patient groups and globally renowned clinicians to launch a report into IPF entitled Inspiration - a new initiative dedicated to raising awareness of IPF,1 a rare disease that is relatively unheard of, frequently misdiagnosed and little understood. [More]
FDA announces availability of $2 million for rare disease research

FDA announces availability of $2 million for rare disease research

The U.S. Food and Drug Administration today announced the availability of $2 million in research grants to fund natural history studies in rare diseases. The aim is to collect data on how specific rare diseases progress in individuals over time so that knowledge can inform and support product development and approval. This will be the first time the FDA will provide funding through its Orphan Products Grants to conduct these types of studies for rare diseases. [More]
Rare disease day focuses on new approach to finding treatment

Rare disease day focuses on new approach to finding treatment

2,200 babies will be born in the UK on 29th February, of which 30 will die of a rare disease before they are five years old. [More]

Clincierge wins 2016 Clinical Informatics News Best Practices award

Clincierge, a provider of specialized services that increase the patient-centricity of clinical drug development and help remove burdens associated with trial participation, today announced that they have been named a winner of the 2016 Clinical Informatics News Best Practices awards. [More]
Influenza virus can able to circumvent the body's immune system

Influenza virus can able to circumvent the body's immune system

Influenza is able to mask itself, so that the virus is not initially detected by our immune system. This is the result of new research from Aarhus University. The researchers behind the study hope that the discovery can be used to develop better treatment against influenza and chronic inflammation conditions such as rheumatoid arthritis. [More]
RegeneRx Biopharmaceuticals announces completion of enrollment for Phase 2b/3 dry eye trial in U.S.

RegeneRx Biopharmaceuticals announces completion of enrollment for Phase 2b/3 dry eye trial in U.S.

RegeneRx Biopharmaceuticals, Inc., a clinical-stage drug development company focused on tissue protection, repair and regeneration, today announced that its U.S. joint venture with G-treeBNT, ReGenTree LLC, has completed enrollment of its Phase 2b/3 dry eye trial in the U.S. [More]
New NCCN Guidelines published for Vulvar Cancer

New NCCN Guidelines published for Vulvar Cancer

It is estimated that more than 5,000 cases of Vulvar Cancer were diagnosed in the United States in 2015, and approximately 1,000 women died from the disease[i]. In order to provide comprehensive, up-to-date clinical treatment guidelines for this rare cancer, the National Comprehensive Cancer Network, an alliance of 26 of the nation's leading cancer centers, has published the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Vulvar Cancer. [More]
Novel gene therapy treatment proves safe, effective for factor VII deficiency

Novel gene therapy treatment proves safe, effective for factor VII deficiency

A single injection. That's all someone with a factor VII deficiency would need for a life-long cure, thanks to a new gene therapy treatment developed in a collaboration of researchers at the University of North Carolina and The Children's Hospital of Philadelphia. [More]
UConn-led researchers identify specific gene linked to Hajdu-Cheney syndrome

UConn-led researchers identify specific gene linked to Hajdu-Cheney syndrome

Fragile bones are usually an old person's affliction, but sometimes children are born with them. Now, a team of researchers led by UConn professor Ernesto Canalis has shown in mice that a specific gene can cause the disease, called Hajdu-Cheney syndrome. Overabundant bone-absorbing cells may be causing the disorder's characteristic bone loss, and the researchers hope to find a potential treatment. [More]
Vtesse receives FDA's Breakthrough Therapy designation for VTS-270

Vtesse receives FDA's Breakthrough Therapy designation for VTS-270

Vtesse, Inc. announced today that the U.S. Food and Drug Administration has granted its drug candidate, VTS-270 for treatment of Niemann-Pick Type C1 Disease (NPC), Breakthrough Therapy designation status. Both the FDA and the European Medicines Agency (EMA) had previously granted Orphan Drug status to VTS-270, which is currently in a pivotal Phase 2b/3 clinical trial. [More]
Millendo signs license agreement, secures funding to test new drugs for treatment of endocrine disorders

Millendo signs license agreement, secures funding to test new drugs for treatment of endocrine disorders

Millendo Therapeutics, a University of Michigan startup company, announced an exclusive license agreement with AstraZeneca for the worldwide development and commercialization rights to test a new compound, MLE4901, for the treatment of polycystic ovary syndrome. [More]
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