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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.

29 Jan 2016

Halaven (eribulin mesylate) approved for treatment of liposarcoma

The U.S. Food and Drug Administration today approved Halaven (eribulin mesylate), a type of chemotherapy, for the treatment of liposarcoma (a specific type of soft tissue sarcoma) that cannot be removed by surgery (unresectable) or is advanced (metastatic). This treatment is approved for patients who received prior chemotherapy that contained an anthracycline drug.

28 Jan 2016

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.

27 Jan 2016

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.

20 Jan 2016

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.

18 Jan 2016

IQWiG finds no added benefit for pomalidomide drug in multiple myeloma

Pomalidomide (trade name: Imnovid) has been approved since 2013 for the treatment of multiple myeloma that has returned and is difficult to treat. The drug is an option for adults who have received two or more prior treatment regimens, including treatment with the drugs lenalidomide and bortezomib, and in whom the last treatment had no sufficient effect.

13 Jan 2016

New gene-filtering tool aims to predict disease-causing mutations

Despite their bad reputations, the vast majority of mutations are not harmful. Even in very rare genetic disorders, only one or two genetic variations — out of tens of thousands — is actually the cause of disease. Distinguishing between harmful and harmless mutations has long been a challenge.

13 Jan 2016

U-M research could lead to new ways of fighting X-linked diseases in girls and women

Nearly every girl and woman on Earth carries two X chromosomes in nearly every one of her cells -- but one of them does (mostly) nothing. That's because it's been silenced, keeping most of its DNA locked up and unread like a book in a cage.

11 Jan 2016

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.

6 Jan 2016

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.

6 Jan 2016

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.

5 Jan 2016

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.

4 Jan 2016

Several therapies for hemophilia A, hemophilia B and hemophilia with inhibitors to be launched between 2015-2025

Decision Resources Group forecasts that several therapies will launch for hemophilia A, hemophilia B and hemophilia with inhibitors in the United States and Europe during our 2015-2025 study period.

29 Dec 2015

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.

29 Dec 2015

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.

24 Dec 2015

FDA approves Uptravi tablets to treat adults with pulmonary arterial hypertension

On December 21, the U.S. Food and Drug Administration approved Uptravi (selexipag) tablets to treat adults with pulmonary arterial hypertension (PAH), a chronic, progressive, and debilitating rare lung disease that can lead to death or the need for transplantation.

22 Dec 2015

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.

22 Dec 2015

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.

20 Dec 2015

Soligenix reports positive results from SGX942 Phase 2 trial in patients with head and neck cancer

Soligenix, Inc., a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today positive results in its Phase 2 clinical trial, in which SGX942, a first-in-class Innate Defense Regulator (IDR), at a dose of 1.5 mg/kg, successfully reduced the median duration of severe oral mucositis by 50% in all patients and by 67% in patients receiving the most aggressive chemoradiation therapy (CRT) for treatment of their head and neck cancer.

16 Dec 2015

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.

15 Dec 2015