rare diseases – media

Biontech and Genevant Sciences Join Hands to Roll Out Rare Disease Drugs

In a joint venture, German biopharmaceutical firm BioNTech and Genevant Sciences, a genetic disorder therapeutics maker plan to develop mRNA therapy programmes targeting rare diseases with severely deficient medical attention.
- Source: Pharmaceutical Technology
- 755
- July 16, 2018
biotech Company Insight rare diseases
Woman’s Bone Vanished into Thin Air!

A woman aged 44 years old continuous pain in her left arm and shoulder. The doctors at the Royal Infirmary of Edinburgh in Scotland were unable to figure out the causative factor. Upon taking X-rays, they were astounded to find that the bones were disappearing. The woman was diagnosed with vanishing bone disease (Gorham-Stout disease).
- Source: LiveScience
- 749
- July 12, 2018
bone rare diseases Researches
Therapy Against Progeria

After conducting a clinical trial study at Boston Children’s Hospital, scientists from Brown University recommended that a lonafarnib therapy could increase the survival rate of progeria patients. But the mode of action of lonafarnib is still uncertain.
- Source: Ddu
- 546
- June 7, 2018
progeria rare diseases Researches
Having Back Pain? Know If It Is Cauda Equina Syndrome

When suffering from back pain, especially if it is accompanied by walking difficulty and weakness in lower leg muscles, it is essential to discover the route of the problem. One possible explanation is that it could be symptoms of Cauda Equina syndrome.
- Source: drugdu
- 471
- May 28, 2018
back pain rare diseases Researches
Shire Announces Sale of Oncology Business to Servier for $2.4 Billion

Sale of Oncology business unlocks embedded value within Shire`s portfolio and sharpens focus on core areas reinforcing our leadership in rare diseases
- Source: finance.yahoo
- 665
- April 17, 2018
Company Insights oncology rare diseases
Dova Pharmaceuticals Signs Exclusive Distribution Agreement with Fosun Pharma For Mainland China and Hong Kong

Dova Pharmaceuticals, Inc. (DOVA), a pharmaceutical company focused on acquiring, developing, and commercializing drug candidates for rare diseases where there is a high unmet need, entered into an agreement through its wholly owned subsidiary, AkaRx, Inc., granting Shanghai Fosun Pharmaceutical Industrial Development Co., Ltd., (Fosun Pharma Industrial), a wholly owned subsidiary of Shanghai Fosun Pharmaceutical (Group) Co., Ltd. (Fosun Pharma) (SHA:600196) (HKG:02196), the exclusive development and distribution rights of avatrombopag in mainland China and Hong Kong.
- Source: finance.yahoo
- 653
- March 21, 2018
avatrombopag Company Insight rare diseases
Enzychem Lifesciences Announces the FDA Orphan Drug Designation Granted to EC-18 on Acute Radiation Syndrome (ARS)

Enzychem Lifesciences, announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to EC-18 for the treatment of Acute Radiation Syndrome (ARS).
- Source: china-pharmacy
- 604
- February 1, 2018
ARS EC-18 FDA Orphan Drug Designation rare diseases
Sanofi to acquire Bioverativ for $11.6 billion

Sanofi SA (SAN.FR) said Monday that it will acquire all outstanding shares in Bioverativ Inc. BIVV, -0.61% for $11.6 billion to expand its presence in specialty care and strengthening leadership in rare diseases.
- Source: Marketwatch
- 487
- January 24, 2018
Market Views rare diseases
FDA awards 15 grants for clinical trials to stimulate product development for rare diseases

The U.S. Food and Drug Administration(FDA) announced that it has awarded 15 new clinical trial research grants totaling more than $22 million over the next four years to boost the development of products for patients with rare diseases. These new grants were awarded to principal investigators from academia and industry across the country.
- Source: fda.gov
- 626
- October 9, 2017
Clinical FDA rare diseases Researches

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