Rare Disease News and Research

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September tip sheet from Cedars-Sinai Medical Center

A computer algorithm did a better job than doctors at documenting "red flag" symptoms among patients with gastrointestinal disorders, a Cedars-Sinai study has found, underscoring the potential of the technology to improve patient care.

29 Aug 2015

Practices for expediting corporate market research approval process for faster insights

Delays in the launch and execution of critical market research projects can slow down the decision-making process, resulting in missed opportunities and increased costs. It is, therefore, absolutely critical for biopharmaceutical companies to develop a tactical approach to expedite the market research approval process for quicker and more meaningful insights.

29 Aug 2015

GOSH to improve rare disease diagnosis with adoption of Congenica’s Sapientia technology

Ian was ‘a clumsy child’. He kept bumping into objects and couldn’t see well in the dark. He endured 15 years of tests and misdiagnoses during a lengthy, and sometimes traumatic, diagnostic odyssey.

26 Aug 2015

Novartis receives FDA approval for expanded use of Promacta for younger children with rare blood disorder

Novartis announced today that the US Food and Drug Administration has approved an expanded use for Promacta to include children 1 year of age and older with chronic immune thrombocytopenia who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy.

26 Aug 2015

FDA, EMA accept filing applications for Boehringer Ingelheim's afatinib to treat patients with advanced SCC of the lung

Boehringer Ingelheim today announced that both the U.S. Food and Drug Administration and the European Medicines Agency have accepted filing applications for afatinib for the treatment of patients with advanced squamous cell carcinoma (SCC) of the lung progressing after treatment with first-line chemotherapy.

26 Aug 2015

MTG Biotherapeutics' MTG-201 granted FDA Orphan Drug Designation for malignant mesothelioma treatment

MTG Biotherapeutics, an immuno-oncology company developing novel therapeutics for the treatment of cancer, today announced that the US Food and Drug Administration has granted Orphan Drug Designation for MTG-201, an advanced biologic therapy targeting Dickkopf-3 gene defects in various cancers, for the treatment of malignant mesothelioma.

23 Aug 2015

Landmark 'basket study' shows efficacy of vemurafenib in multiple nonmelanoma BRAFV600-mutated cancers

Researchers from Memorial Sloan Kettering Cancer Center have announced results from the first published basket study, a new form of clinical trial design that explores responses to drugs based on the specific mutations in patients' tumors rather than where their cancer originated.

20 Aug 2015

Study reports results of genetic testing for 22 genetic causes of neonatal diabetes

Over a 10 year period, the time that babies receive genetic testing after being diagnosed with diabetes has fallen from over four years to under two months. Pinpointing the exact genetic causes of sometimes rare forms of diabetes is revolutionising healthcare for these patients.

18 Aug 2015

NCI-MATCH phase II precision medicine trial opens through ECOG-ACRIN

The National Cancer Institute-Molecular Analysis for Therapy Choice (NCI-MATCH) phase II precision medicine trial, known to doctors as trial EAY131, is open through the ECOG-ACRIN Cancer Research Group.

18 Aug 2015

Envarsus XR granted FDA orphan drug designation for prophylaxis of organ rejection in kidney transplant patients

Veloxis Pharmaceuticals A/S, today announced that Envarsus XR was granted Orphan Drug status by the U.S. Food and Drug Administration for prophylaxis of organ rejection in patients who convert from immediate-release tacrolimus. Envarsus XR received marketing authorization from the FDA on July 10, 2015.

14 Aug 2015

Charlotte charity Taylor's Tale inspires new law to create treatments for rare diseases, spur growth in NC

Charlotte charity Taylor's Tale helped inspire a law designed to stimulate the creation of new treatments for rare diseases and spur economic development in North Carolina.

13 Aug 2015

Inhibikase Therapeutics launches observational clinical study in patients who survived PML

Inhibikase Therapeutics, Inc. announces the launch of an observational clinical study in patients who contracted Progressive Multifocal Leukoencephalopathy (PML), the rare and often fatal brain infection that arises as an unintended side effect of some medications used for treatment of Multiple Sclerosis, Crohn's disease, Ulcerative Colitis and certain cancers of the blood and lymph.

13 Aug 2015

Mallinckrodt subsidiary to acquire Therakos for $1.325 billion

Mallinckrodt plc, a leading specialty biopharmaceutical company, and The Gores Group, a global investment firm, announced today that they have entered into a definitive agreement under which a subsidiary of Mallinckrodt will acquire Therakos, Inc. a leading immunotherapy company in a transaction valued at approximately $1.325 billion.

10 Aug 2015

Vtesse reports preliminary results from VTS-270 Phase 1 trial for treatment of Niemann-Pick Disease Type C

Vtesse, Inc. announced preliminary results today from an open-label Phase 1 clinical trial with VTS-270 (a formulation of (2-hydroxypropyl)-beta-cyclodextrin) for treatment of Niemann-Pick Disease Type C (NPC) conducted by researchers at the National Institutes of Health Eunice Kennedy Shriver National Institute of Child Health and Human Development.

6 Aug 2015

European Commission grants orphan drug designation to SGX301 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 that the European Commission, acting on the positive recommendation from the European Medicines Agency Committee for Orphan Medicinal Products (COMP), has granted orphan drug designation to synthetic hypericin (the active pharmaceutical ingredient in SGX301) for the treatment of cutaneous T-cell lymphoma (CTCL), a rare disease and a class of non-Hodgkin's lymphoma, a type of cancer of the white blood cells that are an integral part of the immune system.

4 Aug 2015

Shire acquires Foresight Biotherapeutics for $300 million

Shire plc announced today that it has acquired New York-based, privately held Foresight Biotherapeutics Inc. for $300 million.

3 Aug 2015

BGI to hold 10th International Conference on Genomics in October

The 10th International Conference on Genomics (ICG-10) will be held by BGI from October 22-25, 2015, in Shenzhen, China.

31 Jul 2015

Lupin signs strategic asset purchase agreement with Temmler

Pharma Major Lupin Limited today announced that it has entered into a strategic asset purchase agreement with TEMMLER PHARMA GMBH & CO. KG, a part of the Aenova Group, one of the world's largest pharmaceutical contract manufacturers, to acquire Temmler's specialty product portfolio subject to certain closing conditions.

25 Jul 2015

Melphalan granted FDA Orphan Drug Designation for treatment of cholangiocarcinoma

Delcath Systems, Inc., a specialty pharmaceutical and medical device company focused on oncology with an emphasis on the treatment of primary and metastatic liver cancers, announced today that the U.S. Food and Drug Administration Office of Orphan Products Development has granted Orphan Drug Designation for melphalan for the treatment of cholangiocarcinoma.

20 Jul 2015

Novogen's Anisina granted FDA Orphan Drug Designation for neuroblastoma

US-Australian drug discovery company, Novogen Limited, today announced that it has received notification from the U.S. Food and Drug Administration that its chemotherapy candidate drug, Anisina, has been granted Orphan Drug Designation for neuroblastoma.

16 Jul 2015

Rare Disease News and Research

September tip sheet from Cedars-Sinai Medical Center

Practices for expediting corporate market research approval process for faster insights

GOSH to improve rare disease diagnosis with adoption of Congenica’s Sapientia technology

Novartis receives FDA approval for expanded use of Promacta for younger children with rare blood disorder

FDA, EMA accept filing applications for Boehringer Ingelheim's afatinib to treat patients with advanced SCC of the lung

MTG Biotherapeutics' MTG-201 granted FDA Orphan Drug Designation for malignant mesothelioma treatment

Landmark 'basket study' shows efficacy of vemurafenib in multiple nonmelanoma BRAFV600-mutated cancers

Study reports results of genetic testing for 22 genetic causes of neonatal diabetes

NCI-MATCH phase II precision medicine trial opens through ECOG-ACRIN

Envarsus XR granted FDA orphan drug designation for prophylaxis of organ rejection in kidney transplant patients

Charlotte charity Taylor's Tale inspires new law to create treatments for rare diseases, spur growth in NC

Inhibikase Therapeutics launches observational clinical study in patients who survived PML

Mallinckrodt subsidiary to acquire Therakos for $1.325 billion

Vtesse reports preliminary results from VTS-270 Phase 1 trial for treatment of Niemann-Pick Disease Type C

European Commission grants orphan drug designation to SGX301 for treatment of CTCL

Shire acquires Foresight Biotherapeutics for $300 million

BGI to hold 10th International Conference on Genomics in October

Lupin signs strategic asset purchase agreement with Temmler

Melphalan granted FDA Orphan Drug Designation for treatment of cholangiocarcinoma

Novogen's Anisina granted FDA Orphan Drug Designation for neuroblastoma

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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