Rare Disease News and Research

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Bayer, Dimension Therapeutics partner to develop new gene therapy for hemophilia A

Dimension Therapeutics, a company focused on developing novel adeno-associated virus (AAV) gene therapy treatments for rare diseases, today announced it has entered into a collaboration with Bayer HealthCare (Bayer) for the development and commercialization of a novel gene therapy for the treatment of hemophilia A.

23 Jun 2014

Dimension Therapeutics closes $30M financing to advance novel gene therapy programs

Dimension Therapeutics, a gene therapy company focused on developing novel treatments for rare diseases, today announced the closing of a $30 million financing.

23 Jun 2014

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.

20 Jun 2014

Alexion announces that FDA grants ODD to Soliris for Myasthenia Gravis treatment

Alexion Pharmaceuticals (Nasdaq:ALXN) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Soliris® (eculizumab) for the treatment of patients with Myasthenia Gravis (MG), a rare, debilitating neurologic disorder caused by uncontrolled complement activation.

17 Jun 2014

Houston clinic finds ways to limit drug industry influence

Doctors are becoming wary of the appearance that drug makers' gifts can influence their prescription choices. Meanwhile, the federal government serves notice that it wants to find a way to preserve a regulation that would allow hospitals to get discounts on orphan drugs.

17 Jun 2014

Research to assess breastfeeding knowledge and confidence of Canadian physicians

The results of a national research project to assess breastfeeding knowledge, confidence, beliefs, and attitudes of Canadian physicians are available today in the Journal of Human Lactation.

13 Jun 2014

Isis Pharmaceuticals begins ISIS-DMPK Rx Phase 1 study for treatment of Myotonic Dystrophy Type 1

Isis Pharmaceuticals, Inc. announced today that it has initiated a Phase 1 study for ISIS-DMPKRx. Isis earned a $14 million milestone payment from Biogen Idec associated with this achievement.

9 Jun 2014

2014 DIA patient fellows illustrate the power of patient advocacy

The 2014 DIA patient fellows illustrate the power of patient advocacy. A Parkinson's disease patient is the driving force in the success of clinical trials, recruiting patients and transporting them to research sites while saving researchers thousands of dollars.

9 Jun 2014

Ocugen's OCU100 gets FDA orphan drug designation for treatment of retinitis pigmentosa

Ocugen, Inc. and the University of Colorado today announced exclusive license agreements that allow for Ocugen to continue developing two drug candidates for the treatment for ophthalmology indications, and that one of the assets, OCU100, recombinant lens epithelium derived growth factor 1-326 (LEDGF1-326), received orphan-drug status from the U.S. Food and Drug Administration for treatment of retinitis pigmentosa (RP), a rare eye disease.

6 Jun 2014

NORD's letter to FDA raises concerns on rare diseases

In a letter submitted today to the U.S. Food and Drug Administration (FDA), the National Organization for Rare Disorders (NORD) urged Commissioner Margaret Hamburg to give serious consideration to the concerns of the rare disease community when setting policy regarding official names for biologics, including biosimilars.

5 Jun 2014

Cerenis’ SAMBA and MODE Phase II Studies Meet Primary Endpoints for Treatment of FPHA, HoFH

Cerenis Therapeutics, the biopharmaceutical company, today announced that two of its Phase II studies, SAMBA and MODE (Modifying Orphan Disease Evaluation), with CER-001, an engineered human apoA-I-containing pre-β HDL mimetic, met their primary clinical endpoints in patients with Familial Primary Hypoalphalipoproteinemia (FPHA) and Homozygous Familial Hypercholesterolemia (HoFH), respectively.

2 Jun 2014

New software tool helps identify genetic mutations linked to increased risk for cancer

Researchers at The University of Texas MD Anderson Cancer Center and other institutions have applied a newly developed software tool to identify genetic mutations that contribute to a person's increased risk for developing common, complex diseases, such as cancer.

31 May 2014

FDA grants Angiochem’s ANG1005 fast track and orphan drug designation for treatment of glioblastoma multiforme

Angiochem announced today that the Food & Drug Association (FDA) has granted both orphan drug and fast track designation to ANG1005 a novel paclitaxel-peptide drug conjugate leveraging the low density lipoprotein receptor-related protein 1 (LRP-1) pathway to cross the blood-brain barrier (BBB) and enter cancer, for the treatment of glioblastoma multiforme (GBM).

31 May 2014

Newron Pharmaceuticals submits safinamide NDA to FDA

Newron Pharmaceuticals S.p.A. ("Newron"), a research and development company focused on novel CNS and pain therapies, and its partner Zambon S.p.A., an international pharmaceutical company strongly committed to the CNS therapeutic area with a long experience in respiratory disease therapies, woman care and primary care, announce that the New Drug Application (NDA) for safinamide was submitted today to the US Food and Drug Administration (FDA).

30 May 2014

Abiogen Pharma, Lee's Pharma sign deal to market Neridronic Acid

Abiogen Pharma S.p.A. of Pisa Italy and Lee's Pharm jointly announced the agreement for License, Distribution and Supply for marketing of Attila® (Neridronic Acid) throughout China, Hong Kong, Macau and Taiwan.

27 May 2014

EMA grants Advanced Therapy Medicinal Product classification for TxCell's Col-Treg

TxCell SA, a biotechnology company developing innovative, personalized cell-based immunotherapies using antigen specific regulatory T-cells (Ag-Tregs) for severe chronic inflammatory and autoimmune diseases, announces today that Col-Treg, its second therapeutic candidate from its ASTrIA platform, has been granted Advanced Therapy Medicinal Product (ATMP) classification by the Committee for Advanced Therapies (CAT) of the European Medicines Agency.

22 May 2014

Acceleron, Celgene to give oral presentations on beta-thalassemia, myelodysplastic syndromes at EHA meeting

Acceleron Pharma Inc., a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of novel protein therapeutics for cancer and rare diseases, today reported that Acceleron, Celgene and investigators in the sotatercept and ACE-536 phase 2 clinical trials will give three oral presentations of interim data from ongoing studies in beta-thalassemia and myelodysplastic syndromes as well as a poster presentation of nonclinical data in sickle cell disease at the 19th Annual Congress of the European Hematology Association (EHA) in Milan, Italy from June 12-15, 2014.

22 May 2014

CHEO pediatric surgeon wins TELUS Award for mHealth Innovation

Local pediatric surgeon and entrepreneur Dr. Matthew Bromwich accepted the "TELUS Award for mHealth Innovation" at the 29th Annual CATAAlliance Innovation Awards Gala tonight.

22 May 2014

Aradigm receives QIDP designation for its inhaled antibiotic candidate, Pulmaquin

Aradigm Corporation (the "Company") today announced that the U.S. Food and Drug Administration (FDA) has designated the Company's lead inhaled antibiotic candidate, Pulmaquin®, as a Qualified Infectious Disease Product (QIDP).

20 May 2014

Novel drug for patients with inoperable CTEPH shows improvement in international trial

After a year of being treated with a novel drug, patients with inoperable chronic thromboembolic pulmonary hypertension (CTEPH) and those with persistent or recurrent pulmonary hypertension after an operation for the disease showed sustained improvement in a multicenter, international trial presented at the 2014 American Thoracic Society International Conference.

20 May 2014

Rare Disease News and Research

Bayer, Dimension Therapeutics partner to develop new gene therapy for hemophilia A

Dimension Therapeutics closes $30M financing to advance novel gene therapy programs

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

Alexion announces that FDA grants ODD to Soliris for Myasthenia Gravis treatment

Houston clinic finds ways to limit drug industry influence

Research to assess breastfeeding knowledge and confidence of Canadian physicians

Isis Pharmaceuticals begins ISIS-DMPK Rx Phase 1 study for treatment of Myotonic Dystrophy Type 1

2014 DIA patient fellows illustrate the power of patient advocacy

Ocugen's OCU100 gets FDA orphan drug designation for treatment of retinitis pigmentosa

NORD's letter to FDA raises concerns on rare diseases

Cerenis’ SAMBA and MODE Phase II Studies Meet Primary Endpoints for Treatment of FPHA, HoFH

New software tool helps identify genetic mutations linked to increased risk for cancer

FDA grants Angiochem’s ANG1005 fast track and orphan drug designation for treatment of glioblastoma multiforme

Newron Pharmaceuticals submits safinamide NDA to FDA

Abiogen Pharma, Lee's Pharma sign deal to market Neridronic Acid

EMA grants Advanced Therapy Medicinal Product classification for TxCell's Col-Treg

Acceleron, Celgene to give oral presentations on beta-thalassemia, myelodysplastic syndromes at EHA meeting

CHEO pediatric surgeon wins TELUS Award for mHealth Innovation

Aradigm receives QIDP designation for its inhaled antibiotic candidate, Pulmaquin

Novel drug for patients with inoperable CTEPH shows improvement in international trial

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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