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Mast Therapeutics initiates Phase 3 extension study of vepoloxamer in sickle cell disease

Mast Therapeutics initiates Phase 3 extension study of vepoloxamer in sickle cell disease

Mast Therapeutics, Inc., a clinical-stage biopharmaceutical company leveraging its molecular adhesion and sealant technology (MAST) platform to develop novel therapies for sickle cell disease, heart failure, and arterial disease, today reported that it has initiated an open-label, multicenter Phase 3 extension study of vepoloxamer (MST-188) in sickle cell disease, referred to as "EPIC-E." [More]
Alizé Pharma presents clinical results from the AZP-531 program at the ADA 75th Scientific Sessions

Alizé Pharma presents clinical results from the AZP-531 program at the ADA 75th Scientific Sessions

Detailed data will be presented from the two Phase I trials conducted in 76 healthy volunteers and overweight or obese subjects... [More]
CORD calls upon governments, others to join forces to make Canada's Rare Disease Strategy a reality

CORD calls upon governments, others to join forces to make Canada's Rare Disease Strategy a reality

Today, on Parliament Hill, the Canadian Organization for Rare Disorders released Canada's Rare Disease Strategy and called upon the federal, provincial and territorial governments along researchers, healthcare providers, educators, employers, and the patient community to join forces to make the Strategy a reality. [More]
Lafora rare disease could be aggravated by oxidative stress

Lafora rare disease could be aggravated by oxidative stress

Neurodegenerative Lafora disease usually becomes apparent through seizures during adolescence and puberty and occurs as a consequence of defects in glycogen metabolism and in the cellular mechanisms that are responsible for its disposal. Researchers at the University of Valencia have led a study in which they propose that Lafora rare disease could be aggravated by oxidative stress. [More]
Stanford University professor named recipient of 2015 ECOG-ACRIN Young Investigator Award

Stanford University professor named recipient of 2015 ECOG-ACRIN Young Investigator Award

Heather Wakelee, MD, is the recipient of the 2015 ECOG-ACRIN Young Investigator Award, the ECOG-ACRIN Cancer Research Group announced today. Dr. Wakelee is associate professor of medicine in the Division of Oncology at the Stanford University School of Medicine in Stanford, Calif. [More]
Partners congratulate researchers on forming new biotechnology company

Partners congratulate researchers on forming new biotechnology company

The Children's Hospital of Eastern Ontario, The Ottawa Hospital, the University of Ottawa and McMaster University congratulate its researchers on forming Turnstone Biologics Inc., a biotechnology company focused on developing new treatments for cancer that harness the patient's own immune system. [More]
Tuberous Sclerosis Alliance to observe fourth annual TSC Global Awareness Day

Tuberous Sclerosis Alliance to observe fourth annual TSC Global Awareness Day

On May 15, the Tuberous Sclerosis Alliance (TS Alliance) will join tuberous sclerosis complex (TSC) organizations around the world to observe the fourth annual TSC Global Awareness Day. [More]
Stem cell disease model reveals how tumor suppressor may drive bone cancer

Stem cell disease model reveals how tumor suppressor may drive bone cancer

Using induced pluripotent stem cells (iPSCs), a team led by Mount Sinai researchers has gained new insight into genetic changes that may turn a well known anti-cancer signaling gene into a driver of risk for bone cancers, where the survival rate has not improved in 40 years despite treatment advances. [More]
Prolong Pharmaceuticals' SANGUINATE granted FDA Orphan Drug Designation for SCD treatment

Prolong Pharmaceuticals' SANGUINATE granted FDA Orphan Drug Designation for SCD treatment

Prolong Pharmaceuticals, LLC, a biopharmaceutical company dedicated to developing products for the treatment of anemias, cancers and their debilitating comorbidities, announced today that the U.S. Food and Drug Administration has granted Orphan Drug Designation for its flagship product SANGUINATE for the treatment of Sickle Cell Disease (SCD). [More]

CentoMD: World's largest genetic mutation database for rare diseases

CENTOGENE AG announces the launch of the world's largest genetic mutation database for rare diseases, CentoMD. CentoMD is a comprehensive and unique repository of genetic variants, including a huge number of unpublished pathogenic variants gathered from patients worldwide. [More]
Shire announces availability of Natpara (parathyroid hormone) for injection in U.S.

Shire announces availability of Natpara (parathyroid hormone) for injection in U.S.

Shire plc today announced that Natpara (parathyroid hormone) for injection is now available in the United States. The U.S. Food and Drug Administration approved Natpara as an adjunct to calcium and vitamin D to control hypocalcemia in patients with hypoparathyroidism on January 23, 2015. [More]
Patient with severe Alzheimer's shows promising benefits during treatment with Bryostatin drug

Patient with severe Alzheimer's shows promising benefits during treatment with Bryostatin drug

Researchers at the Blanchette Rockefeller Neurosciences Institute and the Marshall University Joan C. Edwards School of Medicine announced their findings from a new study entitled, "PSEN1 Variant in a Family with Atypical AD." An Alzheimer patient with very severe disease, genetically confirmed to have a known variant of PSEN1, showed promising benefits during treatment with the drug Bryostatin 1. [More]
First randomized, controlled trial of pharmacotherapy in PCD patients now in place

First randomized, controlled trial of pharmacotherapy in PCD patients now in place

Primary ciliary dyskinesia (PCD) is a rare, genetic respiratory tract disorder leading to progressive pulmonary dysfunction. It exhibits many problems typical for orphan diseases: delayed diagnosis, limited evidence-based knowledge etc. The European Union is funding BESTCILIA (Better Experimental Screening and Treatment for Primary Ciliary Dyskinesia) to introduce uniform guidelines for PCD treatment and diagnosis across Europe. [More]
Confluence pharmaceuticals signs co-development and marketing agreement with AOP orphan for fragile X syndrome drug

Confluence pharmaceuticals signs co-development and marketing agreement with AOP orphan for fragile X syndrome drug

Confluence is a biopharmaceutical company focused on developing therapeutic treatments for Fragile X Syndrome and Autism Spectrum Disorders. AOP is an Austrian based company specializing in the development and marketing of medicines for rare and complex diseases. [More]

Everyday Health announces acquisition of Cambridge BioMarketing Group

Everyday Health, Inc., a leading provider of digital health and wellness solutions, today acquired Cambridge BioMarketing Group, LLC, the leader in strategic launch and marketing solutions for orphan and rare disease products. [More]
Stempeutics' Stempeucel drug receives ATMP classification from EMA for treatment of TAO

Stempeutics' Stempeucel drug receives ATMP classification from EMA for treatment of TAO

Stempeutics Research, a group company of Manipal Education and Medical Group and a Joint Venture with Cipla Group, announced today that the European Medicines Agency has granted Advanced Therapy Medicinal Product classification for its novel stem cell drug 'Stempeucel' which will be used for the treatment of Thromboangiitis Obliterans (TAO). [More]
Selten Pharma's SPI-026 granted FDA Orphan Drug Designation for treatment of PAH

Selten Pharma's SPI-026 granted FDA Orphan Drug Designation for treatment of PAH

Selten Pharma, Inc., a privatively held biopharmaceutical company focused on the development and commercialization of therapies for the treatment of rare diseases, announced today that its lead compound tacrolimus (SPI-026) has been granted Orphan Drug Designation by the U.S. Food & Drug Administration for the treatment of pulmonary arterial hypertension (PAH). [More]
FDA approves CHOLBAM (cholic acid) for treatment of bile acid synthesis disorders

FDA approves CHOLBAM (cholic acid) for treatment of bile acid synthesis disorders

Asklepion Pharmaceuticals, LLC. today announced that the U.S. Food and Drug Administration (FDA) approved CHOLBAM (cholic acid) as a once-daily treatment for cholic acid deficiency in bile acid synthesis disorders due to single enzyme defects. [More]
Researchers discover gene associated with congenital anomaly of urinary tract

Researchers discover gene associated with congenital anomaly of urinary tract

An interdisciplinary team of researchers under the direction of the University of Bonn Hospital have discovered a gene which is associated with a rare congenital anomaly of the urinary tract called classic bladder exstrophy. [More]
Young immigrants to Canada at greater risk of developing IBD

Young immigrants to Canada at greater risk of developing IBD

The younger a person is when they immigrate to Canada, the higher their risk of developing inflammatory bowel disease (IBD), and its major subtypes Crohn's disease and ulcerative colitis, according to a study by researchers at the University of Ottawa, the Institute for Clinical Evaluative Sciences and the Children's Hospital of Eastern Ontario. [More]
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