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.
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).
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.
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.
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.
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.
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.
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