Rare Disease News and Research

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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.

5 Aug 2014

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.

29 Jul 2014

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.

25 Jul 2014

Lyfebulb, Immune collaborate to increase awareness of bullous pemphigoid

Lyfebulb, the International Pemphigus Pemphigoid Foundation (IPPF) and Immune Pharmaceuticals Inc., announced a collaboration to increase awareness of unmet needs in treating bullous pemphigoid last night at an event attended by more than sixty patients, physicians, scientists and other interested parties.

24 Jul 2014

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.

24 Jul 2014

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).

23 Jul 2014

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

22 Jul 2014

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).

18 Jul 2014

Baxter International acquires drug candidate developed to treat sickle cell disease

A drug candidate developed by researchers at the NIH's National Center for Advancing Translational Sciences and its collaborators to treat sickle cell disease has been acquired by Baxter International's BioScience business.

11 Jul 2014

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.

10 Jul 2014

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.

8 Jul 2014

CASI's ENMD-2076 gets FDA Orphan Drug designation for HCC treatment

CASI Pharmaceuticals, Inc., a clinical-stage pharmaceutical company developing therapeutics for the treatment of cancer and other diseases, announced today that its orally-active, Aurora A/angiogenic kinase inhibitor, ENMD-2076, has received Orphan Drug designation from the U.S. Food and Drug Administration for the treatment of hepatocellular carcinoma (HCC).

7 Jul 2014

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.

3 Jul 2014

Galderma's trifarotene molecule gets FDA Orphan Drug Designation for treatment of congenital ichthyosis

Galderma announces that the U.S. FDA granted Orphan Drug Designation status for the company's trifarotene molecule for the treatment of congenital ichthyosis. Based on this decision, Galderma plans to implement a clinical development plan, reinforcing its commitment to exploring new treatment options for rare diseases, as well as meeting the needs of all patients with skin diseases over the course of their lives.

2 Jul 2014

Researchers develop new methods for analysis of clinical studies in small patient groups

Six to eight per cent of the EU population - equivalent to as many as 36 million people - suffer from one of the 5,000 to 8,000 known rare diseases. Both for the research of new treatments for rare diseases, as well as personalised medicine and the development of medicines for children, the patient groups available are often only small.

1 Jul 2014

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.

25 Jun 2014

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.

25 Jun 2014

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).

23 Jun 2014

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.

23 Jun 2014

Rare Disease News and Research

Computer simulation forecasts favorable trends in eradicating hepatitis C

Hepatitis C could become a rare disease by 2036

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

EMA recommends full marketing approval for IMBRUVICA in the European Union

Lyfebulb, Immune collaborate to increase awareness of bullous pemphigoid

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

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

Researchers collaborate to tackle rare diseases

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

Baxter International acquires drug candidate developed to treat sickle cell disease

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

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

CASI's ENMD-2076 gets FDA Orphan Drug designation for HCC treatment

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

Galderma's trifarotene molecule gets FDA Orphan Drug Designation for treatment of congenital ichthyosis

Researchers develop new methods for analysis of clinical studies in small patient groups

CHEO, ONF jointly launch first comprehensive pediatric concussion guidelines

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

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

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

Achieving and maintaining ISO/IEC 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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