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Adolescents in Sweden are at increased risk for type 1 diabetes than previously thought

New research published in Diabetologia (the journal of the European Association for the Study of Diabetes) suggests that Sweden-the country already thought to have the second highest prevalence of type 1 diabetes in the world-could have 2-3 times more adolescents and young adults with type 1 diabetes than previously estimated.

7 Apr 2014

Jain Foundation's dysferlinopathy clinical study exceeds original goal of 150 patients

The Jain Foundation is delighted to announce that 193 patients have been recruited into the Clinical Outcome Study for Dysferlinopathy (COS), exceeding the original goal of 150 patients.

2 Apr 2014

FDA grants orphan drug designation to ERYTECH’s lead product for AML treatment

ERYTECH Pharma, a French biopharmaceutical company that develops innovative treatments for acute leukemia and other oncology indications with unmet medical needs, announces that its lead product GRASPA®/ERY-ASP has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of Acute Myeloid Leukemia (AML), an aggressive form of blood cancer.

2 Apr 2014

Researchers develop new screening tool to help diagnose obstructive sleep apnea in children

Clinical investigators at the Children's Hospital of Eastern Ontario (CHEO) have developed a new screening tool to help diagnose obstructive sleep apnea in children. Their findings are published in Pediatric Pulmonology.

2 Apr 2014

Second Sight's Argus II Retinal Prosthesis System selected for Forfait Innovation reimbursement

Second Sight Medical Products Inc. announces today that the Argus II Retinal Prosthesis System, the world’s first approved device intended to restore some functional vision for people suffering from blindness due to outer retinal degenerations, has become the first recipient of the French Government national healthcare reimbursement program entitled ‘Forfait Innovation’.

1 Apr 2014

Machaon Diagnostics' aHUS Genetic Panel confirms atypical Hemolytic Uremic Syndrome in 48 hours

Machaon Diagnostics today announced the availability of its "aHUS Genetic Panel" (patent pending) to genetically confirm atypical Hemolytic Uremic Syndrome (aHUS) with a turnaround time of 48 hours. The 48 hour timeframe is a dramatic advancement over other approaches which offer results in 4-13 weeks.

1 Apr 2014

CytRx’s aldoxorubicin receives European orphan medicinal product designation for treatment of advanced soft tissue sarcomas

CytRx Corporation, a biopharmaceutical research and development company specializing in oncology, today announced that aldoxorubicin has received orphan medicinal product designation from the European Commission for the treatment of advanced soft tissue sarcomas. The designation is to encourage the development of drugs which may provide significant benefit to patients suffering from rare diseases.

31 Mar 2014

FDA grants orphan drug designation for Kite Pharma's cancer immunotherapy product

Kite Pharma, Inc., a clinical-stage biotechnology company focused on developing engineered autologous T cell therapy (eACT) products for cancer, today announced that the U.S. Food and Drug Administration Office of Orphan Products Development granted orphan drug designation for the Company's lead investigational therapy, an autologous engineered T cell product that targets CD19 expression on B cell malignancies, for the treatment of diffuse large B cell lymphoma (DLBCL).

31 Mar 2014

Research findings help explain rare genetic disorder that causes immunodeficiency syndrome

IRCM researchers led by Javier M. Di Noia, PhD, uncovered a new function of AID, a crucial enzyme for the immune response. The discovery, recently published by the scientific journal Proceedings of the National Academy of Sciences, helps explain a rare genetic disorder that causes an immunodeficiency syndrome.

28 Mar 2014

FDA grants PMA for Pinnacle's Optiguide DCYL700 Fiber Optic Diffuser Series flexible fiber

Concordia Healthcare Corp. announced today that its subsidiary Pinnacle Biologics, Inc., a biopharmaceutical research and development company specializing in rare diseases, was granted U.S. Food and Drug Administration premarket supplemental approval (PMA) for its Optiguide DCYL700 Fiber Optic Diffuser Series flexible fiber.

26 Mar 2014

Dr. Jean-Philippe Chaput receives IJO New Faculty Award for research on obesity

At the International Congress on Obesity held in Kuala Lumpur, Malaysia last week, Dr. Jean-Philippe Chaput from the Healthy Active Living and Obesity research group at the Children's Hospital of Eastern Ontario Research Institute was awarded the International Journal of Obesity (IJO) New Faculty Award for Population Science and Public Health Research.

26 Mar 2014

Otsuka obtains regulatory approval in Japan for pharmacological treatment of ADPKD

Otsuka Pharmaceutical Co., Ltd. today announced it has become the first company in the world to obtain regulatory approval for a pharmacological treatment of autosomal dominant polycystic kidney disease (ADPKD). Samsca (generic name: tolvaptan) has been approved in Japan in 7.5-mg and 15-mg tablet forms for extended use for the additional indication of ADPKD. Also, the new dosage form of 30-mg Samsca tablets has received approval for the indication of ADPKD.

24 Mar 2014

FDA finds medical device recalls nearly doubled in 10 years

The Wall Street Journal says a report to be released today will spotlight the increasing number of problems.

24 Mar 2014

First Edition: March 21, 2014

Today's early morning highlights from the major news organizations, including a new examination of Ezekiel Emanuel's book about the crafting of the health law and President Obama's latest push to boost enrollment.

21 Mar 2014

Scientists discover cellular pathway that may prevent onset of Type 2 diabetes

Scientists at the Children's Hospital of Eastern Ontario (CHEO) Research Institute have discovered a cellular pathway that is responsible for keeping blood sugar levels low in obese or pre-diabetic people, and may prevent the onset of Type 2 diabetes.

20 Mar 2014

USTAR Center for Genetic Discovery to integrate genome data with patient care

The USTAR Center for Genetic Discovery is partnering with California based Omicia, Inc, to make analyzing a patient's genome as routine as performing a blood test.

19 Mar 2014

Researchers identify two novel genes associated with development of rare, aggressive blood cancer

Researchers have identified two novel cancer genes that are associated with the development of a rare, highly aggressive, cancer of blood vessels. These genes may now act as markers for future treatments and explain why narrowly targeted therapies that are directed at just one target fail.

17 Mar 2014

Three innovative tools to detect, test and initiate personalized drug treatment for seizures

A team of researchers from the National Institutes of Health, Emory University and Cedars-Sinai - specialists in identifying and treating very rare diseases - used three innovative tools to detect a previously unknown gene mutation, test potential therapies in the lab, and initiate personalized drug treatment for a boy with a lifelong history of uncontrollable seizures that caused significant impact on his cognitive and social development.

12 Mar 2014

Patient with autistic behavior experiences improvements in mental health after liver transplant

A patient with a rare metabolic disease that causes liver failure and autistic behavior experienced significant improvements in both her physical and mental health after receiving a liver transplant, according to a new case report published in the American Journal of Transplantation.

12 Mar 2014

Longtime allies team up to create Penn Medicine/CHOP Friedreich's Ataxia Center of Excellence

Three longtime allies have joined forces to create the new Penn Medicine/CHOP Friedreich's Ataxia Center of Excellence.

7 Mar 2014

Rare Disease News and Research

Adolescents in Sweden are at increased risk for type 1 diabetes than previously thought

Jain Foundation's dysferlinopathy clinical study exceeds original goal of 150 patients

FDA grants orphan drug designation to ERYTECH’s lead product for AML treatment

Researchers develop new screening tool to help diagnose obstructive sleep apnea in children

Second Sight's Argus II Retinal Prosthesis System selected for Forfait Innovation reimbursement

Machaon Diagnostics' aHUS Genetic Panel confirms atypical Hemolytic Uremic Syndrome in 48 hours

CytRx’s aldoxorubicin receives European orphan medicinal product designation for treatment of advanced soft tissue sarcomas

FDA grants orphan drug designation for Kite Pharma's cancer immunotherapy product

Research findings help explain rare genetic disorder that causes immunodeficiency syndrome

FDA grants PMA for Pinnacle's Optiguide DCYL700 Fiber Optic Diffuser Series flexible fiber

Dr. Jean-Philippe Chaput receives IJO New Faculty Award for research on obesity

Otsuka obtains regulatory approval in Japan for pharmacological treatment of ADPKD

FDA finds medical device recalls nearly doubled in 10 years

First Edition: March 21, 2014

Scientists discover cellular pathway that may prevent onset of Type 2 diabetes

USTAR Center for Genetic Discovery to integrate genome data with patient care

Researchers identify two novel genes associated with development of rare, aggressive blood cancer

Three innovative tools to detect, test and initiate personalized drug treatment for seizures

Patient with autistic behavior experiences improvements in mental health after liver transplant

Longtime allies team up to create Penn Medicine/CHOP Friedreich's Ataxia Center of Excellence

Sensing a Healthier Future

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

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