Shape Therapeutics inks gene therapy deal with Roche worth up to USD 3 Billion Seattle biotech firm Shape Therapeutics has signed a deal potentially exceeding USD 3 billion with pharma giant Roche ...
Rare diseases, collectively, can not be considered rare at all. According to the National Institute of Health, approximately 30 million Americans that makeup 10% of the total American population, are affected by one of the 7,000 known rare diseases. However, the challenges manifested by the Rare patient population ...
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Primary hyperoxaluria (PH) is a rare autosomal genetic form of Hyperoxaluria, a condition that leads to the excessive urinary excretion of oxalate. Till now, three distinct hereditary enzymatic deficiencies have been identified as a cause of PH, namely, PH type 1 (PH1), type 2 (PH2), and type 3 (PH3), with PH1 as t...
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The necessity of a robust Angelman syndrome pipeline is indispensable at the current moment because of a shortage of any approved therapy option available in the market. The Angelman Syndrome current treatment landscape consists of symptomatic therapy options worth USD 330.2 million in 2017. Treatment entire...
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TLX66 Meets Study Objectives, Holding Out in AL Amyloidosis Telix Pharmaceuticals reported the results from the TRALA trial (Targeted Radiotherapy for AL Amyloidosis), a Phase I/IIa trial evaluating the safety and toxicity of TLX66 as the sole bone marrow conditioning agent prior to autologous hematopoietic stem...
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Hearing loss is observed to be the most common sensory disorder. Hearing loss is found to be an etiologically diverse condition that is accompanied by various disease-related complications and major clinical, social, and implications affecting the quality of life. Approximately 50% to 60% of hearing loss patients a...
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Abingworth Achieves Financial Close, Raises USD 582 Million Abingworth, a London-based venture capital firm, has announced the closing of its new Clinical Co-Development Fund 2 (ACCD 2) worth USD 582 million, however, the fund was aiming to gather USD $350 million. Not long ago, the company also closed ...
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Alagille syndrome (ALGS), also referred to as Alagille–Watson syndrome, is a rare autosomal dominant condition. Mutation in JAG1 or NOTCH2 gene causes the condition to occur, but most of the individuals’ mutation in the JAG1 gene is noted. The symptoms vary in patients, and the severity of the disease ranges from m...
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Alpha1-antitrypsin deficiency (AATD) was first identified by Carl-Bertil Laurell and Sten Eriksson (Laurell and Eriksson 1963) in 1963, who discovered a link between low plasma serum AAT levels and pulmonary emphysema symptoms. With time as the understanding of the genetic abnormalities grew, AAT deficiency c...
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Asher Bio raises USD 55 Million in Series A financing Asher Biotherapeutics, a biotechnology company developing exactly targeted immunotherapies for cancer and other diseases, raised USD 55 Million in Series A financing. Third Rock Ventures led the round with participation from Boxer Capital of Tavistock Grou...
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It was assumed initially that Alzheimer’s Disease was a rare condition, but now it is considered t.....
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Market intelligence (MI) is described as the information or the data derived by a company from the m.....
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Pulmonary Arterial Hypertension (PAH) is a rare, chronic, and progressive form of Pulmonary Hyperten.....
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A rare autosomal recessive disorder, Familial chylomicronemia syndrome (FCS), affects 1 in 1 to 2 mi.....
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Halitosis is an oral health problem caused by volatile molecules which are formed because of patholo.....
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Rheumatoid Arthritis (RA) is a chronic, inflammatory autoimmune disease that leads to progressive an.....
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The American Society of Clinical Oncology (ASCO) is one of the largest and most respected conferences in the field of oncology. Held annually, this conference brings together researchers, physicians, and other healthcare professionals from around the world to discuss the latest advances in cancer research, diagnosis, and treatment.
