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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

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.

12 Dec 2015

Alecensa (alectinib) approved for treatment of people with advanced ALK-positive NSCLC

The U.S. Food and Drug Administration today approved Alecensa (alectinib) to treat people with advanced (metastatic) ALK-positive non-small cell lung cancer (NSCLC) whose disease has worsened after, or who could not tolerate treatment with, another therapy called Xalkori (crizotinib).

11 Dec 2015

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.

10 Dec 2015

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.

9 Dec 2015

FDA approves recombinant von Willebrand factor for treating bleeding episodes in adults with VWD

The U.S. Food and Drug Administration today approved Vonvendi, von Willebrand factor (Recombinant), for use in adults 18 years of age and older who have von Willebrand disease (VWD). Vonvendi is the first FDA-approved recombinant von Willebrand factor, and is approved for the on-demand (as needed) treatment and control of bleeding episodes in adults diagnosed with VWD.

8 Dec 2015

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.

8 Dec 2015

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.

8 Dec 2015

Predictors of cerebral cavernous malformation outcomes pinpointed

A meta-analysis of individual patient data has identified risk factors for intracerebral haemorrhage in patients with cerebral cavernous malformations.

4 Dec 2015

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.

1 Dec 2015

FDA grants approval for Empliciti (elotuzumab) to treat patients with multiple myeloma

Today the U.S. Food and Drug Administration granted approval for Empliciti (elotuzumab) in combination with two other therapies to treat people with multiple myeloma who have received one to three prior medications.

30 Nov 2015

Mexico Healthcare and Life Sciences Report 2015 released by Pharmaboardroom

Pharmaboardroom releases its new 98-page Mexico Healthcare and Life Sciences Report 2015. It offers companies, investors, policymakers, and stakeholders with an interest in healthcare, pharmaceuticals, or life sciences insights into the second largest pharma and healthcare market in Latin America, a country of over 122 million people.

30 Nov 2015

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.

28 Nov 2015

Health insurance coverage for transgender people is cost-effective, study finds

A new analysis led by the Johns Hopkins Bloomberg School of Public Health suggests that while most U.S. health insurance plans deny benefits to transgender men and women for medical care necessary to transition to the opposite sex, paying for sex reassignment surgery and hormones is actually cost-effective.

25 Nov 2015

Ninlaro (ixazomib) approved to treat people with multiple myeloma

Today the U.S. Food and Drug Administration granted approval for Ninlaro (ixazomib) in combination with two other therapies to treat people with multiple myeloma who have received at least one prior therapy.

21 Nov 2015

Health experts urge lawmakers, regulators to close loopholes in Orphan Drug Act

Health experts at Johns Hopkins Medicine are calling on lawmakers and regulators to close loopholes in the Orphan Drug Act they claim give drug companies millions of dollars in unintended and misplaced subsidies and tax breaks and fuel skyrocketing medication costs.

20 Nov 2015

Rockefeller University researchers discover molecule that helps guide axons during CNS development

During embryonic development, billions of neurons nimbly reposition themselves within the brain and spinal cord, and connect branches to form the neural circuits that ultimately control our movements, perception, and memory. Scientists have long sought to understand the driving forces in this meticulously choreographed dance, in which each cell seems to know precisely where to be to at any given time, and with what other cells it must link.

20 Nov 2015

FDA grants accelerated approval for Darzalex to treat patients with multiple myeloma

Today the U.S. Food and Drug Administration granted accelerated approval for Darzalex (daratumumab) to treat patients with multiple myeloma who have received at least three prior treatments. Darzalex is the first monoclonal antibody approved for treating multiple myeloma.

17 Nov 2015

MedUni Vienna researchers discover genetic cause of a rare disease

Researchers at MedUni Vienna have discovered the genetic cause of a rare disease characterized by life-threatening "liver crises" in early childhood and subsequent manifestation of neurological symptoms, such as neuropathy and ataxia (a movement disorder), when they reach school age.

15 Nov 2015

FDA grants accelerated approval for Tagrisso to treat patients with advanced NSCLC

Today, the U.S. Food and Drug Administration granted accelerated approval for an oral medication to treat patients with advanced non-small cell lung cancer (NSCLC). Tagrisso (osimertinib) is now approved for patients whose tumors have a specific epidermal growth factor receptor (EGFR) mutation (T790M) and whose disease has gotten worse after treatment with other EGFR-blocking therapy.

14 Nov 2015

Rare Disease News and Research

Discovery could pave way for targeted therapies to treat penile cancer

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

Alecensa (alectinib) approved for treatment of people with advanced ALK-positive NSCLC

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

Scientists reveal key links between childhood obesity and healthy living behaviours

FDA approves recombinant von Willebrand factor for treating bleeding episodes in adults with VWD

Kanuma approved as first treatment for patients with LAL deficiency

CTTV launches new web platform for identifying therapeutic targets

Predictors of cerebral cavernous malformation outcomes pinpointed

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

FDA grants approval for Empliciti (elotuzumab) to treat patients with multiple myeloma

Mexico Healthcare and Life Sciences Report 2015 released by Pharmaboardroom

Mallinckrodt sells CMDS business to Guerbet for $270 million

Health insurance coverage for transgender people is cost-effective, study finds

Ninlaro (ixazomib) approved to treat people with multiple myeloma

Health experts urge lawmakers, regulators to close loopholes in Orphan Drug Act

Rockefeller University researchers discover molecule that helps guide axons during CNS development

FDA grants accelerated approval for Darzalex to treat patients with multiple myeloma

MedUni Vienna researchers discover genetic cause of a rare disease

FDA grants accelerated approval for Tagrisso to treat patients with advanced NSCLC

Why use light scattering to analyze proteins and viral vectors?

Changing the landscape of R&D to build clinical success from the ground up

Sensing a Healthier Future

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