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S.No. Product Name Actions
1 Beta Thalassemia Treatment - Insights Into the Challenges & Solutions Visit Post
2 The Evolving Neurofibromatosis Treatment Landscape Visit Post
3 Boston Scientific's MODULAR CRM System; Restore Medical's ContraBand clinical trial result; BD acquires Venclose; Roche differentiates mutations in Omicron; Linus's StrandDx-ASD; HelioLiver launches liquid biopsy test Visit Post
4 Aslan Pharma - IQVIA collaboration; Reata's kidney drug bardoxolone; Alzheimer’s Disease treatment update; Recordati to acquire EUSA Visit Post
5 Avrobio’s Gene Therapy for Cystinosis Treatment: A New Hope to Overcome the Existing Medical Unmet Needs Visit Post
6 Neurent Medical’s NEUROMARK; Brain Navi NaoTrac received European CE Mark approval; Theradaptive receives Breakthrough Medical Device Designation; HAGAR GWave awarded Breakthrough Device Designation; Fujifilm launches ColoAssist PRO Visit Post
7 The Evolved Gene Therapy for Hemophilia Visit Post
8 An in-depth Assessment of the Top Drugs Launched by Leading Global Companies in the First Half of 2021 (H1) Visit Post
9 von Willebrand disease (VWD) – Misdiagnosis Still Prevalent! Visit Post
10 Discover How Learning Disability Treatment Landscape is Evolving with the Emergence of Digital Assistant Technologies Visit Post
11 Pfizer tightens DMD trial criteria over safety concerns; Kytopen raises $30M; Eli Lilly taps Reify's Care Access; Vensana Capital raises $325M Visit Post
12 Boston Scientific pays $336M; Boehringer Ingelheim acquires Abexxa Biologics; Oncology, RNA biotech 858 Therapeutics inks $60M Series A; Novartis acquires Arctos Medical Visit Post
13 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
14 Charcot-Marie-Tooth Disease Market Size Expects Growth Owing to Increasing Prevalence, Influx of Pharma Companies and Novel Therapies Visit Post
15 Duchenne Muscular Dystrophy Market: What's More Beyond Exon-Skipping Therapies? Visit Post
16 Shape Therapeutics-Roche's Deal; AllStripes Raises $50M; Datavant-Real Chemistry's Partnership; BlueWillow's Nasal Vaccine Visit Post
17 Von Hippel-Lindau Market Space Expected to Expand at a CAGR 8.3% by 2030 Owing to Increasing Prevalence and Emerging Therapies Visit Post
18 Primary Hyperoxaluria Market Size Observes Growth with a Substantial CAGR Visit Post
19 Angelman syndrome Pipeline: Unmet needs call for a robust pipeline Visit Post
20 TLX66 in AL Amyloidosis; Takeda’s Dengue Shot; Innovent’s Cholangiocarcinoma Clinical Trial; Another Failed NASH Drug Visit Post
21 Who is Going to be a Trendsetter in the Hereditary Deafness Market? Visit Post
22 Abingworth & Alebund's Finacial Closing; Pfizer/BioNTech COVID-19 Vaccine Expanded Use; Biogen and Capsigen Deal Visit Post
23 Rising Awareness, Key Companies, Emerging Therapies? What is Shaping the Alagille Syndrome Market Visit Post
24 Alpha-1 Antitrypsin Deficiency: Symptoms, Treatments and Key Companies in the Market Visit Post
25 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
26 Hemophilia B Market: How Pipeline Therapies are Transforming the Treatment Hemisphere? Visit Post
27 A Royal Disease: Hemophilia Visit Post
28 Emerging Research Continues To Transform Erythropoietic Protoporphyria Market Outlook Visit Post
29 Biohaven's Troriluzole Failure; Daiichi/ AZ's Enhertu; Fujifilm & Manufacturing Spree; J&J's Darzalex Faspro Visit Post
30 Angelman Syndrome Market: Insights Into The Recent Late-Stage Drug Failures And Novel Approaches To Treating This Rare Neurogenetic Disorder Visit Post
31 21 of the most common questions about Epilepsy Visit Post
32 Ever-evolving Market Dynamics of Epilepsy – a silver lining! Visit Post
33 Argininosuccinic Aciduria Market Awaits Innovative Treatment Expected To Launch In The Next Decade Visit Post
34 Which pipeline therapy has the potential to revolutionize the Down Syndrome Market? Visit Post
35 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
36 Unfolding The Folds Of Transthyretin Visit Post
37 A Complex Chromosomal Abberation Called Adrenoleukodystrophy Visit Post
38 Is the cure for Huntington’s disease on the horizon? Visit Post
39 Angelman syndrome: A Rare Genetic Disorder Visit Post
40 GBT, Syros; Takeda, Cevance collaborates; IND application to ZN-c3 Visit Post
41 Fragile X Syndrome Market Visit Post
42 UREA CYCLE DISORDER - A LOST ENZYME may lead to FATAL RISKS Visit Post
43 Novel treatment approval promises a better future for Sickle cell disease patients Visit Post
44 How Gene therapy is changing the Beta-thalassemia Treatment outlook? Visit Post
45 AbbVie Sells bonds to Fund Allergan takeover Visit Post
46 Beta-Thalassemia Pharmacological Therapies Visit Post
47 FDA declines the approval of Lipocine’s testosterone drug; Novartis Sandoz to buy Aspen Pharmacare Japanese Unit Visit Post
48 The growing size of Sanfilippo syndrome Market: Drivers and Barriers Visit Post
49 Down Syndrome Awareness Month Visit Post
50 Batten Disease Market: Barriers and Growth Opportunities Visit Post
51 Flagship Pioneering launches Omega Therapeutics; EU nod to Vitrakvi; Bluebird Bio reveals positive results of Starbeam clinical trial Visit Post
52 How Spinal Muscular Atrophy Therapies transform market? Visit Post
53 Niemann–Pick disease type C Therapeutics Market Visit Post
54 CHPM’s positive approval for 13 Medicines; NICE approval for AstraZeneca’s Olaparib; Exact Sciences to buy Genomic Health Visit Post
55 Recursion Pharmaceuticals nets USD 121M; Bayer's MRI agent gets a nod Visit Post
56 The era of Gene therapy, and the Billion Dollar tag Visit Post
57 World Sickle Cell Day Visit Post
58 Origins of the depressive behaviour in Huntington's disease Visit Post
59 What is causing a rise in Autism rate? Visit Post
60 Meet the World's most expensive drug: Zolgensma Visit Post
61 Antitrypsin Deficiency Pipeline Visit Post
62 Unmet Needs in the treatment of Antitrypsin deficiency Visit Post
63 AstraZeneca-Daiichi’s results; Novartis remunerates; Pfizer to acquire Therachon; Gilead & Goldfinch Bio collaboration Visit Post
64 Spinal Muscular Atrophy Market Visit Post
65 Spinal Muscular Atrophy: Current and Emerging Therapies Visit Post
66 Spinal Muscular Atrophy Market is expected to augment at a CAGR of 10.42% Visit Post
67 Autism Spectrum Disorder Visit Post
68 Can Spinal Muscular Atrophy be treated by Protease Inhibitors? Visit Post
69 Boys are more likely to have autism! Visit Post
70 Approach for Cystic Fibrosis; An alternative for Statins; Oral medications not safe; Carbon monoxide poisoning treatment gets approval Visit Post
71 Biogen buys Nightstar; Pacira acquires Myoscience; Horizon Pharma announces pricing; STAT with Slingshot Insights; Epizyme announces Equity Offering Visit Post
72 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
73 The Business Cocktail Visit Post
74 Peloton raises $150M; BiomX raises $32M; Abbott and Novo collaborate; Blaze Bioscience nets $5M Visit Post
75 Notizia Visit Post
76 Artificial Intelligence face-scanning app spots signs of rare genetic disorders Visit Post
77 Notizia Visit Post
78 Innovent Biologics reaps; Jeff Aronin’s lead; Alexion gives out; Stoke Therapeutics receives Visit Post
79 Notizia Visit Post
80 Notizia Visit Post
81 Kalydeco got approved; Opdivo for SCLC; FDA approved for pain relief; GC4419 obviates Visit Post
82 Regulatory Visit Post
83 Personalized Medicine Approach: An Upholding Paradigm for a Promising and futuristic Patient Centric Health Care Visit Post
84 Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) – a lesser developed window Visit Post
85 Hematopoietic Stem Cell Transplantation Visit Post
86 Notizia Visit Post
87 Phase 1 data of CYC065; Lysogene delays phase 3 study; Genetic disorders Diagnosis; PD-1 resistance in melanoma patients Visit Post
88 Notizia Visit Post
89 Notizia Visit Post
90 Transthyretin Amyloidosis (ATTR) Competitive Landscape, Market Insights, Epidemiology and Market Visit Post
91 Symdeko’s approval; Erytech target solid tumors; Oncologie snapped bavituximab; Merck delivers Visit Post
92 Familial Amyloid Polyneuropathy – An uncommon medical condition Visit Post
93 Notizia Visit Post
94 Hereditary Angioedema (HAE) – Active drug pipeline Visit Post
95 Systemic Lupus Erythematosus (SLE) – Market Is Expected to Grow with Upcoming Therapies Visit Post
96 Gaucher Disease: “Rare Disorder with High Unmet Needs” Visit Post
97 Bubble Boy Syndrome (ADA-SCID) - A Rare Immunodeficiency Disorder Visit Post
98 Wiskott - Aldrich syndrome – a rare X-linked disorder Visit Post
99 Metachromatic Leukodystrophy (MLD): A Rare Indication with great unmet medical need Visit Post
100 Business cocktail Visit Post
101 Can-Fite Receives Payment; Leafbuyer Technologies and Denver Post’s Cannabist; EPX-300 & JOTROL received Designation; New treatment Approved for adults Visit Post
102 Friedrich's ataxia: Rare disease Visit Post
103 Stada’s €5.3B; Pfizer takes 30 cents; GSK, Exscientia ink deal; Trillium hunts for partners; Clementia files for $115M IPO Visit Post
104 Hemophilia: A Lifelong Genetic Disorder Visit Post
105 Samsung biosim nod sets; Lilly's med hits; BioMarin sees pricing; Spinraza scores; Klick creates Visit Post
106 Drug shortage; Fujifilm invests; Cobra Biologics to pump $19M; The world's most pricey drugs Visit Post
107 Hemophilia A- Market Scenario Visit Post
108 PMV Raises; Argos Shares Plummet; Cidara Flunks in; Sarepta Sells to Gilead; Novartis and NHS Ayrshire & Arran launch Visit Post
109 Fragile X syndrome: X-linked mental retardation and macroorchidism Visit Post
110 Systemic Lupus Erythematosus: An autoimmune disease Visit Post
111 Obsessive-Compulsive Disorder: Emerging Therapies Visit Post
112 Novartis Buys; FDA approves; Mayne Pharma Launches; Oxford Genetics Receives Grant Visit Post
113 Autism study finds early intervention has lasting effects Visit Post
114 Sarepta’s Unusual Journey Visit Post
115 Do we need to revise human genetics? Visit Post
116 FDA Approves Sarepta’s Muscular Dystrophy Drug after Months of Debate Visit Post
117 Spinal Muscular Atrophy Emerging therapies Visit Post
118 Duchenne Muscular Dystrophy Market Visit Post
119 DelveInsight’s Genetic disorders based Gene Therapy Reports Visit Post