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

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). [More]
Enzyme therapy may prevent skeletal abnormalities associated with neurofibromatosis type-1

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. [More]
Scientists identify mechanism by which inherited neurological disease causes muscle weakness in men

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. [More]
Idera collaborates with TMA to advance new potential treatment approach for myositis

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. [More]
Computer simulation forecasts favorable trends in eradicating hepatitis C

Computer simulation forecasts favorable trends in eradicating hepatitis C

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. [More]
Hepatitis C could become a rare disease by 2036

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. [More]
Researchers confirm for the first time that achalasia is autoimmune in origin

Researchers confirm for the first time that achalasia is autoimmune in origin

Achalasia is a rare disease - it affects 1 in 100,000 people - characterized by a loss of nerve cells in the esophageal wall. [More]
EMA recommends full marketing approval for IMBRUVICA in the European Union

EMA recommends full marketing approval for IMBRUVICA in the European Union

Pharmacyclics, Inc. today announced that the Committee for Medicinal Products for Human Use of the European Medicines Agency issued a positive opinion recommending the granting of full marketing approval for IMBRUVICA (ibrutinib) in the European Union. [More]
Zydelig gets FDA approval for treatment of patients with three types of blood cancers

Zydelig gets FDA approval for treatment of patients with three types of blood cancers

The U.S. Food and Drug Administration today approved Zydelig (idelalisib) to treat patients with three types of blood cancers. [More]
Shire, ArmaGen partner to develop AGT-182 drug for treatment of Hunter syndrome

Shire, ArmaGen partner to develop AGT-182 drug for treatment of Hunter syndrome

Shire plc, the global specialty biopharmaceutical company, and ArmaGen, a US privately held biotechnology company, today announced a worldwide licensing and collaboration agreement for AGT-182, an investigational enzyme replacement therapy (ERT) for the potential treatment of both the central nervous system (CNS) and somatic manifestations in patients with Hunter syndrome (MPS II). [More]
Researchers collaborate to tackle rare diseases

Researchers collaborate to tackle rare diseases

Support from a network of leading researchers across Europe specialised in a rare auto-immune disease with unmet medical needs could help test several novel treatments [More]
FDA approves Ruconest for treatment of acute attacks in adolescent patients with HAE

FDA approves Ruconest for treatment of acute attacks in adolescent patients with HAE

The U.S. Food and Drug Administration yesterday approved Ruconest, the first recombinant C1-Esterase Inhibitor product for the treatment of acute attacks in adult and adolescent patients with hereditary angioedema (HAE). [More]
Sigma-Tau Rare Disease seeks EMA approval for use of Oncaspar for ALL treatment

Sigma-Tau Rare Disease seeks EMA approval for use of Oncaspar for ALL treatment

Sigma Tau Pharma Ltd. (hereinafter referred to as Sigma-Tau Rare Disease), today announced the submission of an application to the European Medicines Agency for use of its pegylatedL-asparaginase, Oncaspar (pegaspargase) for the treatment of Acute Lymphoblastic Leukaemia (ALL) as part of a multi-agent chemotherapeutic regimen. [More]
Fabry disease awareness: an interview with Dr. Hartmann Wellhoefer, Head of Medical Affairs, Rare Disease, Shire

Fabry disease awareness: an interview with Dr. Hartmann Wellhoefer, Head of Medical Affairs, Rare Disease, Shire

Lysosomes are subcellular organelles that are present in most cells, with the major exception of red blood cells. [More]
Beleodaq gets FDA approval for treatment of patients with peripheral T-cell lymphoma

Beleodaq gets FDA approval for treatment of patients with peripheral T-cell lymphoma

The U.S. Food and Drug Administration today approved Beleodaq (belinostat) for the treatment of patients with peripheral T-cell lymphoma (PTCL), a rare and fast-growing type of non-Hodgkin lymphoma (NHL). The action was taken under the agency's accelerated approval program. [More]
CHEO, ONF jointly launch first comprehensive pediatric concussion guidelines

CHEO, ONF jointly launch first comprehensive pediatric concussion guidelines

Pediatric emergency medicine researchers at the Children's Hospital of Eastern Ontario together with the Ontario Neurotrauma Foundation today launch the first comprehensive pediatric concussion guidelines. [More]
Patients with Cushing’s syndrome experience significant weight loss while taking oral medication

Patients with Cushing’s syndrome experience significant weight loss while taking oral medication

Sinai Hospital of Baltimore endocrinologist, Henry G. Fein, M.D., today presented new research showing that patients with Cushing's syndrome, a rare disease that can lead to extreme weight gain, type 2 diabetes, high blood pressure and psychological issues, experienced significant, sustained weight loss while taking oral medication daily over a number of years to manage symptoms of the disease. [More]
Aradigm begins Pulmaquin Phase III study for treatment of non-CF BE

Aradigm begins Pulmaquin Phase III study for treatment of non-CF BE

Aradigm Corporation ("Aradigm" or the "Company") today announced the dosing of the first patient in the ORBIT-4 (Once-daily Respiratory Bronchiectasis Inhalation Treatment) Phase III pivotal clinical trial of Aradigm's proprietary formulation of inhaled ciprofloxacin (Pulmaquin®) for the treatment of non-cystic fibrosis bronchiectasis (non-CF BE). [More]
Cincinnati Children’s, Alexion Pharmaceuticals establish ‘Rare Disease Innovation Fund’

Cincinnati Children’s, Alexion Pharmaceuticals establish ‘Rare Disease Innovation Fund’

Alexion Pharmaceuticals, Inc. and Cincinnati Children's Hospital Medical Center today announced the establishment of a collaboration and fund for the advancement of research in rare disease. [More]
Insurers face difficult cost and coverage calculations regarding gene-sequencing tests

Insurers face difficult cost and coverage calculations regarding gene-sequencing tests

Aimee Robeson just wants an answer. Her son, Christian, was born in 2010 with multiple, mysterious syndromes that leave him unable to speak, chew, or walk on his own. [More]