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1 Boston Scientific pays $336M; Boehringer Ingelheim acquires Abexxa Biologics; Oncology, RNA biotech 858 Therapeutics inks $60M Series A; Novartis acquires Arctos Medical Visit Post
2 FDA’s New Drug Application to ALS Drug; Approval to BeiGene’s Brukinsa; Roche, Temedica Forges Digital Companionship, Shares of Sage Therapeutics Rises Visit Post
3 Charcot-Marie-Tooth Disease Market Size Expects Growth Owing to Increasing Prevalence, Influx of Pharma Companies and Novel Therapies Visit Post
4 Duchenne Muscular Dystrophy Market: What's More Beyond Exon-Skipping Therapies? Visit Post
5 Shape Therapeutics-Roche's Deal; AllStripes Raises $50M; Datavant-Real Chemistry's Partnership; BlueWillow's Nasal Vaccine Visit Post
6 Von Hippel-Lindau Market Space Expected to Expand at a CAGR 8.3% by 2030 Owing to Increasing Prevalence and Emerging Therapies Visit Post
7 Primary Hyperoxaluria Market Size Observes Growth with a Substantial CAGR Visit Post
8 Angelman syndrome Pipeline: Unmet needs call for a robust pipeline Visit Post
9 TLX66 in AL Amyloidosis; Takeda’s Dengue Shot; Innovent’s Cholangiocarcinoma Clinical Trial; Another Failed NASH Drug Visit Post
10 Who is Going to be a Trendsetter in the Hereditary Deafness Market? Visit Post
11 Abingworth & Alebund's Finacial Closing; Pfizer/BioNTech COVID-19 Vaccine Expanded Use; Biogen and Capsigen Deal Visit Post
12 Rising Awareness, Key Companies, Emerging Therapies? What is Shaping the Alagille Syndrome Market Visit Post
13 Alpha-1 Antitrypsin Deficiency: Symptoms, Treatments and Key Companies in the Market Visit Post
14 Asher Bio raises $55M; Roche halts Huntington's phase 3 trial; Novartis' radioligand hits goal in phase 3; EpimAb raises $120M in Series C Visit Post
15 Hemophilia B Market: How Pipeline Therapies are Transforming the Treatment Hemisphere? Visit Post
16 A Royal Disease: Hemophilia Visit Post
17 Emerging Research Continues To Transform Erythropoietic Protoporphyria Market Outlook Visit Post
18 Biohaven's Troriluzole Failure; Daiichi/ AZ's Enhertu; Fujifilm & Manufacturing Spree; J&J's Darzalex Faspro Visit Post
19 Angelman Syndrome Market: Insights Into The Recent Late-Stage Drug Failures And Novel Approaches To Treating This Rare Neurogenetic Disorder Visit Post
20 21 of the most common questions about Epilepsy Visit Post
21 Ever-evolving Market Dynamics of Epilepsy – a silver lining! Visit Post
22 Argininosuccinic Aciduria Market Awaits Innovative Treatment Expected To Launch In The Next Decade Visit Post
23 Which pipeline therapy has the potential to revolutionize the Down Syndrome Market? Visit Post
24 FDA's Ok to Roche's Oral SMA Therapy; Roche's Etrolizumab Mixed Results in Ulcerative Colitis; Secura receives rights to Copiktra from Verastem Visit Post
25 Unfolding The Folds Of Transthyretin Visit Post
26 A Complex Chromosomal Abberation Called Adrenoleukodystrophy Visit Post
27 Is the cure for Huntington’s disease on the horizon? Visit Post
28 Angelman syndrome: A Rare Genetic Disorder Visit Post
29 GBT, Syros; Takeda, Cevance collaborates; IND application to ZN-c3 Visit Post
30 Fragile X Syndrome Market Visit Post
32 Novel treatment approval promises a better future for Sickle cell disease patients Visit Post
33 How Gene therapy is changing the Beta-thalassemia Treatment outlook? Visit Post
34 AbbVie Sells bonds to Fund Allergan takeover Visit Post
35 Beta-Thalassemia Pharmacological Therapies Visit Post
36 FDA declines the approval of Lipocine’s testosterone drug; Novartis Sandoz to buy Aspen Pharmacare Japanese Unit Visit Post
37 The growing size of Sanfilippo syndrome Market: Drivers and Barriers Visit Post
38 Down Syndrome Awareness Month Visit Post
39 Batten Disease Market: Barriers and Growth Opportunities Visit Post
40 Flagship Pioneering launches Omega Therapeutics; EU nod to Vitrakvi; Bluebird Bio reveals positive results of Starbeam clinical trial Visit Post
41 How Spinal Muscular Atrophy Therapies transform market? Visit Post
42 Niemann–Pick disease type C Therapeutics Market Visit Post
43 CHPM’s positive approval for 13 Medicines; NICE approval for AstraZeneca’s Olaparib; Exact Sciences to buy Genomic Health Visit Post
44 Recursion Pharmaceuticals nets USD 121M; Bayer's MRI agent gets a nod Visit Post
45 The era of Gene therapy, and the Billion Dollar tag Visit Post
46 World Sickle Cell Day Visit Post
47 Origins of the depressive behaviour in Huntington's disease Visit Post
48 What is causing a rise in Autism rate? Visit Post
49 Meet the World's most expensive drug: Zolgensma Visit Post
50 Antitrypsin Deficiency Pipeline Visit Post
51 Unmet Needs in the treatment of Antitrypsin deficiency Visit Post
52 AstraZeneca-Daiichi’s results; Novartis remunerates; Pfizer to acquire Therachon; Gilead & Goldfinch Bio collaboration Visit Post
53 Spinal Muscular Atrophy Market Visit Post
54 Spinal Muscular Atrophy: Current and Emerging Therapies Visit Post
55 Spinal Muscular Atrophy Market is expected to augment at a CAGR of 10.42% Visit Post
56 Autism Spectrum Disorder Visit Post
57 Can Spinal Muscular Atrophy be treated by Protease Inhibitors? Visit Post
58 Boys are more likely to have autism! Visit Post
59 Approach for Cystic Fibrosis; An alternative for Statins; Oral medications not safe; Carbon monoxide poisoning treatment gets approval Visit Post
60 Biogen buys Nightstar; Pacira acquires Myoscience; Horizon Pharma announces pricing; STAT with Slingshot Insights; Epizyme announces Equity Offering Visit Post
61 ColdZyme inactivates viruses; Med for rare genetic disease; Common oral bacteria accelerate Colon cancer; CEPI and CureVac to develop an mRNA vaccine; Celastrol’s effectiveness Visit Post
62 The Business Cocktail Visit Post
63 Peloton raises $150M; BiomX raises $32M; Abbott and Novo collaborate; Blaze Bioscience nets $5M Visit Post
64 Notizia Visit Post
65 Artificial Intelligence face-scanning app spots signs of rare genetic disorders Visit Post
66 Notizia Visit Post
67 Innovent Biologics reaps; Jeff Aronin’s lead; Alexion gives out; Stoke Therapeutics receives Visit Post
68 Notizia Visit Post
69 Notizia Visit Post
70 Kalydeco got approved; Opdivo for SCLC; FDA approved for pain relief; GC4419 obviates Visit Post
71 Regulatory Visit Post
72 Personalized Medicine Approach: An Upholding Paradigm for a Promising and futuristic Patient Centric Health Care Visit Post
73 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) – a lesser developed window Visit Post
74 Hematopoietic Stem Cell Transplantation Visit Post
75 Notizia Visit Post
76 Phase 1 data of CYC065; Lysogene delays phase 3 study; Genetic disorders Diagnosis; PD-1 resistance in melanoma patients Visit Post
77 Notizia Visit Post
78 Notizia Visit Post
79 Transthyretin Amyloidosis (ATTR) Competitive Landscape, Market Insights, Epidemiology and Market Visit Post
80 Symdeko’s approval; Erytech target solid tumors; Oncologie snapped bavituximab; Merck delivers Visit Post
81 Familial Amyloid Polyneuropathy – An uncommon medical condition Visit Post
82 Notizia Visit Post
83 Hereditary Angioedema (HAE) – Active drug pipeline Visit Post
84 Systemic Lupus Erythematosus (SLE) – Market Is Expected to Grow with Upcoming Therapies Visit Post
85 Gaucher Disease: “Rare Disorder with High Unmet Needs” Visit Post
86 Bubble Boy Syndrome (ADA-SCID) - A Rare Immunodeficiency Disorder Visit Post
87 Wiskott - Aldrich syndrome – a rare X-linked disorder Visit Post
88 Metachromatic Leukodystrophy (MLD): A Rare Indication with great unmet medical need Visit Post
89 Business cocktail Visit Post
90 Can-Fite Receives Payment; Leafbuyer Technologies and Denver Post’s Cannabist; EPX-300 & JOTROL received Designation; New treatment Approved for adults Visit Post
91 Friedrich's ataxia: Rare disease Visit Post
92 Stada’s €5.3B; Pfizer takes 30 cents; GSK, Exscientia ink deal; Trillium hunts for partners; Clementia files for $115M IPO Visit Post
93 Hemophilia: A Lifelong Genetic Disorder Visit Post
94 Samsung biosim nod sets; Lilly's med hits; BioMarin sees pricing; Spinraza scores; Klick creates Visit Post
95 Drug shortage; Fujifilm invests; Cobra Biologics to pump $19M; The world's most pricey drugs Visit Post
96 Hemophilia A- Market Scenario Visit Post
97 PMV Raises; Argos Shares Plummet; Cidara Flunks in; Sarepta Sells to Gilead; Novartis and NHS Ayrshire & Arran launch Visit Post
98 Fragile X syndrome: X-linked mental retardation and macroorchidism Visit Post
99 Systemic Lupus Erythematosus: An autoimmune disease Visit Post
100 Obsessive-Compulsive Disorder: Emerging Therapies Visit Post
101 Novartis Buys; FDA approves; Mayne Pharma Launches; Oxford Genetics Receives Grant Visit Post
102 Autism study finds early intervention has lasting effects Visit Post
103 Sarepta’s Unusual Journey Visit Post
104 Do we need to revise human genetics? Visit Post
105 FDA Approves Sarepta’s Muscular Dystrophy Drug after Months of Debate Visit Post
106 Spinal Muscular Atrophy Emerging therapies Visit Post
107 Duchenne Muscular Dystrophy Market Visit Post
108 DelveInsight’s Genetic disorders based Gene Therapy Reports Visit Post