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FDA grants multiple Orphan Drug Designations for CytRx's aldoxorubicin

CytRx Corporation, a biopharmaceutical research and development company specializing in oncology, today announced that the U.S. Food and Drug Administration has granted multiple Orphan Drug Designations for the Company's lead drug candidate, aldoxorubicin, in three indications: glioblastoma multiforme (GBM), small cell lung cancer and ovarian cancer.

29 Sep 2014

UCLA research may lead to novel treatment for PH1

Research by UCLA biochemists may lead to a new treatment - or even a cure - for PH1, a rare and potentially deadly genetic kidney disease that afflicts children. Their findings also may provide important insights into treatments for Parkinson's disease, Alzheimer's disease and other degenerative diseases.

27 Sep 2014

Yale University researchers study potential new treatment to reverse effects of pulmonary fibrosis

Yale University researchers are studying a potential new treatment that reverses the effects of pulmonary fibrosis, a respiratory disease in which scars develop in the lungs and severely hamper breathing.

23 Sep 2014

Rosuvastatin promotes bone growth in mice with achondroplasia symptoms

Skeletal dysplasia is a group of rare diseases that afflict skeletal growth through abnormalities in bone and cartilage. Its onset hits at the fetal stage and is caused by genetic mutations.

18 Sep 2014

Synthetic Biologics' SYN-005 antibody gets FDA Orphan Drug designation for Pertussis treatment

Synthetic Biologics, Inc., a biotechnology company developing novel anti-infective biologic and drug programs targeting specific pathogens that cause serious infections and diseases, announced today that the U.S. Food and Drug Administration has granted Orphan Drug designation to the Company's proprietary SYN-005 monoclonal antibody (mAb) combination for the treatment of Pertussis, more commonly known as whooping cough.

15 Sep 2014

Mayo Clinic and IBM plan to pilot Watson to begin research studies in cancer

Mayo Clinic and IBM today announced plans to pilot Watson, the IBM cognitive computer, to match patients more quickly with appropriate clinical trials, beginning with research studies in cancer. A proof-of-concept phase is currently underway.

9 Sep 2014

Soligenix acquires novel orphan drug candidate for treatment of CTCL

Soligenix, Inc., a late-stage biopharmaceutical company developing products that address unmet medical needs in the areas of inflammation, oncology and biodefense, announced today the acquisition of a novel orphan drug candidate, known as SGX301 (synthetic hypericin).

8 Sep 2014

Researchers discover genetic factors involved in development of early-onset ulcerative colitis

UCLA researchers were part of a team that has discovered the interplay of several genetic factors that may be involved in the development of early-onset ulcerative colitis, a severe type of inflammatory bowel disease.

4 Sep 2014

Concordia's subsidiary signs collaboration deal with Orphan Canada for PHOTOFRIN

Concordia Healthcare Corp., a diverse healthcare company focused on legacy pharmaceutical products, orphan drugs, and medical devices for the diabetic population, today announced that its subsidiary, Pinnacle Biologics, Inc., a biopharmaceutical research and development company specializing in rare diseases, has signed a collaboration agreement for PHOTOFRIN® with Orphan Canada, a Toronto-based specialty pharmaceutical company that in‐licenses therapies for rare disorders and specialty medicines within Canada.

2 Sep 2014

Study: One in every 200 Ontarians diagnosed with inflammatory bowel disease

One in every 200 Ontarians has been diagnosed with inflammatory bowel disease (IBD), with the number of people living with the disease increasing by 64 per cent between 1999 and 2008, according to a study by researchers at the Institute for Clinical Evaluative Sciences, the Children's Hospital of Eastern Ontario, and the Ottawa Hospital Research Institute.

29 Aug 2014

New Coalition helps prevent abuse, misuse, diversion of ADHD drug

Today is the official launch of a newly formed Coalition of medical, mental health, higher education, student and pharmaceutical organizations that will work together to help prevent misuse, abuse and diversion of ADHD prescription stimulant medication. The Coalition will initially focus its efforts on college students.

28 Aug 2014

DNA testing for congenital cataracts can accurately diagnose diseases linked to childhood blindness

Researchers in the United Kingdom have demonstrated that advanced DNA testing for congenital cataracts can quickly and accurately diagnose a number of rare diseases marked by childhood blindness, according to a study published online today in Ophthalmology, the journal of the American Academy of Ophthalmology.

22 Aug 2014

Study shows bryostatin plays key role in slowing or reversing Alzheimer's disease

Neurotrope, Inc. (OTCQB: NTRP) today announced that results from a new study, entitled, "PKCe Deficits in Alzheimer's Disease Brains and Skin Fibroblasts," published in the recent edition of the peer-reviewed Journal of Alzheimer's Disease and co-authored by Tapan K. Khan, Ph.D.; Abhik Sen, Ph.D.; Jarin Hongpaisan, Ph.D.; Chol S. Lim, Ph.D.; Thomas J. Nelson, Ph.D., and; Dr. Daniel L. Alkon, each of the Blanchette Rockefeller Neurosciences Institute (BRNI), provide further indication of the role that protein kinase C epsilon (PKCe) may play in the potential treatment of Alzheimer's disease (AD).

19 Aug 2014

Enzyme therapy may prevent skeletal abnormalities associated with neurofibromatosis type-1

An enzyme therapy may prevent skeletal abnormalities associated with the genetic disorder neurofibromatosis type-1, Vanderbilt investigators have discovered.

15 Aug 2014

OncoSynergy's OS2966 receives FDA orphan drug designation for treatment of glioblastoma

OncoSynergy announced today that the FDA Office of Orphan Products Development has granted orphan drug designation for the investigational drug candidate OS2966, a neutralizing anti-CD29 monoclonal antibody, for the treatment of glioblastoma, the most common and deadliest primary adult brain tumor.

12 Aug 2014

Oncothyreon acquires Alpine Biosciences to enable targeted delivery of multiple therapeutic agents

Oncothyreon Inc., a biotechnology company specializing in the development of innovative therapeutic products for the treatment of cancer, today announced that it has acquired Alpine Biosciences, Inc., of Seattle, Washington, a privately held biotechnology company developing protocells, a nanoparticle platform technology designed to enable the targeted delivery of multiple therapeutic agents, including nucleic acids, proteins, peptides and small molecules.

12 Aug 2014

Scientists identify mechanism by which inherited neurological disease causes muscle weakness in men

Researchers at University of California, San Diego School of Medicine have identified the mechanism by which a rare, inherited neurodegenerative disease causes often crippling muscle weakness in men, in addition to reduced fertility.

11 Aug 2014

Idera collaborates with TMA to advance new potential treatment approach for myositis

Idera Pharmaceuticals, Inc., a clinical stage biopharmaceutical company developing nucleic acid therapeutics for patients with cancer and rare diseases, and The Myositis Association (TMA), the only nonprofit organization dedicated to solely serving all patients with inflammatory myopathies, today announced a collaboration to advance a new potential treatment approach for polymyositis and dermatomyositis known as Toll-like receptor (TLR) antagonism.

7 Aug 2014

Computer simulation forecasts favorable trends in eradicating hepatitis C

5 Aug 2014

Hepatitis C could become a rare disease by 2036

Effective new drugs and screening would make hepatitis C a rare disease by 2036, according to a computer simulation conducted by The University of Texas MD Anderson Cancer Center and the University of Pittsburgh Graduate School of Public Health. The results of the simulation are reported in the August 5 edition of the journal Annals of Internal Medicine.