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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]
TWi Biotechnology obtains Rare Disease Drug designation in Taiwan for use of AC-203 to treat EBS

TWi Biotechnology obtains Rare Disease Drug designation in Taiwan for use of AC-203 to treat EBS

TWi Pharmaceuticals, Inc. today announced that its fully owned subsidiary, TWi Biotechnology, Inc., has received the designation of Rare Disease Drug by Taiwan FDA for use of AC-203 to treat Epidermolysis Bullosa Simplex (EBS), and is eligible for applying for coverage under National Health Insurance Administration. [More]
Mallinckrodt enters into definitive agreement to acquire Ikaria

Mallinckrodt enters into definitive agreement to acquire Ikaria

Mallinckrodt plc, a leading global specialty biopharmaceutical company, and Ikaria, Inc. a privately-held critical care company, announced today that they have entered into a definitive agreement under which a subsidiary of Mallinckrodt will acquire Ikaria, Inc. from a Madison Dearborn-led investor group in a transaction valued at approximately $2.3 billion. [More]
CEGIR launches patient contact registry for people with eosinophilic gastrointestinal diseases

CEGIR launches patient contact registry for people with eosinophilic gastrointestinal diseases

To coincide with Rare Disease Day 2015, the Consortium of Eosinophilic Gastrointestinal Disease Researchers today launched a patient contact registry for individuals with eosinophilic gastrointestinal diseases (EGID), a group of rare diseases. [More]
Novel financing technique may unlock funding for developing 'orphan' drugs to treat rare diseases

Novel financing technique may unlock funding for developing 'orphan' drugs to treat rare diseases

A paper published today, "Financing translation: Analysis of the NCATS rare-diseases portfolio" in Science Translational Medicine, demonstrates the potential of a new financing technique to reduce the risk associated with investing in the treatment of new diseases and potentially unlock new levels of funding for developing so-called "orphan" drugs. [More]
CSL Behring donates protein therapies to World Federation of Hemophilia

CSL Behring donates protein therapies to World Federation of Hemophilia

In recognition of Rare Disease Day and as part of its ongoing commitment to the global bleeding disorders community, CSL Behring announced today that it is donating 2 million international units (IUs) of protein therapies to the World Federation of Hemophilia. [More]
Rare Disease Day 2015 raises awareness of challenges faced by rare cancer patients

Rare Disease Day 2015 raises awareness of challenges faced by rare cancer patients

Within the "umbrella" of rare diseases, rare cancer patients confront particular problems. Most rare diseases have an identified genetic origin. In contrast, rare cancers are mainly acquired diseases. [More]
Cipher acquires worldwide rights to three products from Astion

Cipher acquires worldwide rights to three products from Astion

Cipher Pharmaceuticals Inc. today announced it has strengthened its product pipeline by acquiring the worldwide rights to three products from Astion Pharma, a Denmark-based specialty pharmaceutical company. [More]
Tolero's alvocidib receives EMA orphan drug designation for treatment of AML patients

Tolero's alvocidib receives EMA orphan drug designation for treatment of AML patients

Tolero Pharmaceuticals, Inc., a clinical-stage company developing treatments for serious hematological diseases, today announced that the European Medicines Agency has granted orphan drug designation for alvocidib for the treatment of patients with acute myeloid leukemia (AML). [More]
Isis Pharmaceuticals announces positive results from Phase 1 study of ISIS-PKK Rx for HAE treatment

Isis Pharmaceuticals announces positive results from Phase 1 study of ISIS-PKK Rx for HAE treatment

Isis Pharmaceuticals, Inc. announced today positive results from a Phase 1 study with ISIS-PKKRx. In this study, healthy volunteers treated with ISIS-PKKRx achieved dose-dependent reductions of up to 95 percent in prekallikrein, or PKK. ISIS-PKKRx is a RNA-targeted antisense drug designed to inhibit the production of PKK for the prophylactic treatment of hereditary angioedema (HAE). [More]
Shire announces acquisition of Meritage Pharma

Shire announces acquisition of Meritage Pharma

Shire plc and Meritage Pharma, Inc. announced today that Shire has acquired Meritage, a privately-held company, for an upfront fee of $70 million and additional contingent payments based on the achievement of development and regulatory milestones. [More]
Shire announces completion of NPS Pharma acquisition

Shire announces completion of NPS Pharma acquisition

Shire plc announces the successful completion of the tender offer for all of the outstanding shares of NPS Pharmaceuticals, Inc. and the subsequent acquisition of NPS Pharma. [More]

EC grants orphan drug designation to aTyr Pharma's Resolaris for treatment of FSHD

aTyr Pharma, Inc., a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address rare diseases, announced today the European Commission (EC) has granted orphan drug designation to Resolaris for the treatment of facioscapulohumeral muscular dystrophy (FSHD). [More]
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