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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]
New tests could improve diagnosis of two rare childhood diseases

New tests could improve diagnosis of two rare childhood diseases

For the first time, researchers have developed tests that could improve the diagnosis of two rare childhood diseases known as congenital disorders of glycosylation (CDGs) and metachromatic leukodystrophy, and that could even lead to new treatments for CDGs. [More]
Crowdsourcing: the answer for rare disease diagnosis?

Crowdsourcing: the answer for rare disease diagnosis?

Medical crowdsourcing is like the ultimate consult service. A question arises, it is asked on the forum, and you can get answers from multiple specialties within minutes. It is harnessing the brightest minds in one place and using that power to do what we enjoy doing most – helping people. [More]
UAB's Undiagnosed Diseases Program finds answers for mysterious health issues

UAB's Undiagnosed Diseases Program finds answers for mysterious health issues

Stephanie and Christopher Smith have been on a long, difficult and often frustrating journey to find answers for the mysterious health issues of their children. It started 24 years ago when their third child, Gage, was born. Gage, his younger brother Aiden and then sister Mandalynn, now 13, all developed mysterious symptoms that mystified doctors. [More]
Selexipag holds promise for treatment of pulmonary hypertension

Selexipag holds promise for treatment of pulmonary hypertension

Living with pulmonary arterial hypertension is challenging, but the chore of treating the rare heart disease may change following promising clinical trial data to be published in the Dec. 24 issue of the New England Journal of Medicine. [More]
Study reveals new genetic factors linked to age-related macular degeneration

Study reveals new genetic factors linked to age-related macular degeneration

An international study of about 43,000 people has significantly expanded the number of genetic factors known to play a role in age-related macular degeneration (AMD), a leading cause of vision loss among people age 50 and older. [More]
Variant signs worldwide exclusive license and development agreement with L&F Research

Variant signs worldwide exclusive license and development agreement with L&F Research

Variant Pharmaceuticals, Inc., an emerging pharmaceutical company focusing on rare diseases and other specialty conditions with limited or sub-optimal therapeutic options, announced today a worldwide exclusive license and development agreement with L&F Research LLC to advance development of Variant's lead product, VAR 200. [More]
Discovery could pave way for targeted therapies to treat penile cancer

Discovery could pave way for targeted therapies to treat penile cancer

Researchers have identified potential genetic alterations in penile cancer that could pave the way for targeted treatments. [More]
FDA approves Vistogard (uridine triacetate) for emergency treatment of chemotherapy overdose

FDA approves Vistogard (uridine triacetate) for emergency treatment of chemotherapy overdose

The U.S. Food and Drug Administration today approved Vistogard (uridine triacetate) for the emergency treatment of adults and children who receive an overdose of the cancer treatment fluorouracil or capecitabine, or who develop certain severe or life-threatening toxicities within four days of receiving these cancer treatments. [More]
Boehringer Ingelheim begins SENSCIS study to investigate safety, efficacy of nintedanib in patients with SSc-ILD

Boehringer Ingelheim begins SENSCIS study to investigate safety, efficacy of nintedanib in patients with SSc-ILD

Boehringer Ingelheim announced today that the first person has been enrolled in the SENSCIS (Safety and Efficacy ofNintedanib in Systemic SClerosIS) study. [More]
Scientists reveal key links between childhood obesity and healthy living behaviours

Scientists reveal key links between childhood obesity and healthy living behaviours

Scientists from the Children's Hospital of Eastern Ontario Research Institute participated in several peer-reviewed articles that published today in the International Journal of Obesity Supplements. The series (including 16 original contributions) was prepared by the International Study of Childhood Obesity, Lifestyle and the Environment research group, a global collective of leading obesity research experts from 12 countries located on five continents. [More]
Kanuma approved as first treatment for patients with LAL deficiency

Kanuma approved as first treatment for patients with LAL deficiency

Today, the U.S. Food and Drug Administration approved Kanuma (sebelipase alfa) as the first treatment for patients with a rare disease known as lysosomal acid lipase (LAL) deficiency. [More]
CTTV launches new web platform for identifying therapeutic targets

CTTV launches new web platform for identifying therapeutic targets

The Centre for Therapeutic Target Validation (CTTV) has launched a new web platform for life-science researchers that helps them identify therapeutic targets for new and repurposed medicines. Today's launch underscores the CTTV's commitment to share its data openly to benefit the broader scientific community. [More]
Lexicon announces top-line results from TELECAST Phase 3 study of telotristat etiprate

Lexicon announces top-line results from TELECAST Phase 3 study of telotristat etiprate

Lexicon Pharmaceuticals, Inc. announced today that top-line data from its TELECAST Phase 3 study showed results of telotristat etiprate in treating carcinoid syndrome in cancer patients with metastatic neuroendocrine tumors consistent with the clinical benefit observed in its pivotal TELESTAR study. [More]
Mallinckrodt sells CMDS business to Guerbet for $270 million

Mallinckrodt sells CMDS business to Guerbet for $270 million

Mallinckrodt plc, a leading specialty biopharmaceutical company, announced today that it has closed the sale of its global contrast media and delivery systems (CMDS) business to Guerbet in a transaction valued at approximately $270 million. [More]
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